Non-viral therapeutic approaches to ocular diseases: An overview and future directions

Multiplex Genome Engineering Using CRISPR/Cas Systems

Cong, L., Ran, F. A., Cox, D., Lin, S., Barretto, R., Habib, N., Hsu, P. D., Wu, X., Jiang, W., Marraffini, L. A., et al. (2013) · Science · doi:10.1126/science.1231143 · Cited by 15,339 · see citing works

A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine.

Boussif, O., Lezoualc’h, F., Zanta, M. A., Djavaheri‐Mergny, M., Scherman, D., DEMENEIX, B. A., & Behr, J. (1995) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.92.16.7297 · Cited by 6,256 · see citing works

Retinitis pigmentosa

Hartong, D. T., Berson, E. L., & Dryja, T. P. (2006) · The Lancet · doi:10.1016/s0140-6736(06)69740-7 · Cited by 2,924 · see citing works

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.

Cavazzana-Calvo, M., Hacein-Bey, S., Basile, G. D. S., Gross, F., Yvon, E., Nusbaum, P., Selz, F., Hue, C., Certain, S., Casanova, J., et al. (2000) · Science · doi:10.1126/science.288.5466.669 · Cited by 2,703 · see citing works

A short amino acid sequence able to specify nuclear location

Kalderon, D., Roberts, B., Richardson, W. D., & Smith, A. E. (1984) · Cell · doi:10.1016/0092-8674(84)90457-4 · Cited by 2,511 · see citing works

Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles

Davis, M. E., Zuckerman, J. E., Choi, C. H. J., Seligson, D. B., Tolcher, A. W., Alabi, C. A., Yen, Y., Heidel, J. D., & Ribas, A. (2010) · Nature · doi:10.1038/nature08956 · Cited by 2,508 · see citing works

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine · doi:10.1038/nm1358 · Cited by 2,163 · see citing works

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

Maguire, A. M., Simonelli, F., Pierce, E. A., Pugh, E. N., Mingozzi, F., Bennicelli, J., Banfi, S., Marshall, K. A., Testa, F., Surace, E. M., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802315 · Cited by 2,145 · see citing works

Effect of gene therapy on visual function in Leber’s congenital amaurosis

Bainbridge, J. W. B., Smith, A. J., Barker, S. S., Robbie, S., Henderson, R., Balaggan, K., Viswanathan, A., Holder, G. E., Stockman, A., Tyler, N., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802268 · Cited by 1,924 · see citing works

A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

Hacein-Bey-Abina, S., von Kalle, C., Schmidt, M., Le Deist, F., Wulffraat, N., McIntyre, E., Radford, I., Villeval, J.-L., Fraser, C. C., Cavazzana-Calvo, M., et al. (2003) · New England Journal of Medicine · doi:10.1056/nejm200301163480314 · Cited by 1,867 · see citing works

Human TLR9 confers responsiveness to bacterial DNA via species-specific CpG motif recognition

Bauer, S., Kirschning, C. J., Häcker, H., Redecke, V., Hausmann, S., Akira, S., Wagner, H., & Lipford, G. B. (2001) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.161293498 · Cited by 1,527 · see citing works

A very strong enhancer is located upstream of an immediate early gene of human cytomegalovirus

Boshart, M., Weber, F., Jahn, G., DORSCHHLER, K., Fleckenstein, B. W., & Schaffner, W. (1985) · Cell · doi:10.1016/s0092-8674(85)80025-8 · Cited by 1,140 · see citing works

Oral gene delivery with chitosan–DNA nanoparticles generates immunologic protection in a murine model of peanut allergy

Roy, K., Mao, H., Huang, S. K., & Leong, K. W. (1999) · Nature Medicine · doi:10.1038/7385 · Cited by 1,106 · see citing works

Rapid endo‐lysosomal escape of poly(DL‐lactide‐ co glycolide) nanoparticles: implications for drug and gene delivery

Panyam, J., Zhou, W., Prabha, S., Sahoo, S. K., & Labhasetwar, V. D. (2002) · The FASEB Journal · doi:10.1096/fj.02-0088com · Cited by 1,044 · see citing works

Nucleocytoplasmic transport: signals, mechanisms and regulation

Nigg, E. A. (1997) · Nature · doi:10.1038/386779a0 · Cited by 996 · see citing works

Intercellular Trafficking and Protein Delivery by a Herpesvirus Structural Protein

Elliott, G., & O’Hare, P. (1997) · Cell · doi:10.1016/s0092-8674(00)81843-7 · Cited by 975 · see citing works

Retinitis pigmentosa

Hamel, C. (2006) · Orphanet Journal of Rare Diseases · doi:10.1186/1750-1172-1-40 · Cited by 962 · see citing works

Gene Therapy Death Prompts Review of Adenovirus Vector

Marshall, E. (1999) · Science · doi:10.1126/science.286.5448.2244 · Cited by 918 · see citing works

Direct gene transfer with DNA-liposome complexes in melanoma: expression, biologic activity, and lack of toxicity in humans.

Nabel, G. J., Nabel, E. G., Yang, Z., Fox, B. A., Plautz, G. E., Gao, X., Huang, L., Shu, S., Gordon, D., & Chang, A. E. (1993) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.90.23.11307 · Cited by 882 · see citing works

siRNA-mediated gene silencing in vitro and in vivo

Xia, H., Mao, Q., Paulson, H. L., & Davidson, B. L. (2002) · Nature Biotechnology · doi:10.1038/nbt739 · Cited by 873 · see citing works

The sequence 5′-AAUAAA-3′ forms part of the recognition site for polyadenylation of late SV40 mRNAs

Fitzgerald, M. (1981) · Cell · doi:10.1016/0092-8674(81)90521-3 · Cited by 823 · see citing works

The influence of polymer structure on the interactions of cationic polymers with DNA and morphology of the resulting complexes

Tang, M., & Szoka, F. C. (1997) · Gene Therapy · doi:10.1038/sj.gt.3300454 · Cited by 814 · see citing works

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial

MacLaren, R. E., Groppe, M., Barnard, A. R., Cottriall, C. L., Tolmachova, T., Seymour, L., Clark, K. R., During, M. J., Cremers, F. P. M., Black, G. C. M., et al. (2014) · The Lancet · doi:10.1016/s0140-6736(13)62117-0 · Cited by 796 · see citing works

Optimization of the preparation process for human serum albumin (HSA) nanoparticles

Langer, K., Balthasar, S., Vogel, V., Dinauer, N., von Briesen, H., & Schubert, D. (2003) · International Journal of Pharmaceutics · doi:10.1016/s0378-5173(03)00134-0 · Cited by 760 · see citing works

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

Bainbridge, J. W. B., Mehat, M. S., Sundaram, V., Robbie, S. J., Barker, S. E., Ripamonti, C., Georgiadis, A., Mowat, F. M., Beattie, S. G., Gardner, P. J., et al. (2015) · New England Journal of Medicine · doi:10.1056/nejmoa1414221 · Cited by 743 · see citing works

PEGylation significantly affects cellular uptake and intracellular trafficking of non-viral gene delivery particles

Mishra, S., Webster, P. C., & Davis, M. E. (2004) · European Journal of Cell Biology · doi:10.1078/0171-9335-00363 · Cited by 733 · see citing works

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

Cideciyan, A. V., Alemán, T. S., Boye, S. L., Schwartz, S., Kaushal, S., Román, A. J., Pang, J., Sumaroka, A., Windsor, E. A. M., Wilson, J. M., et al. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0807027105 · Cited by 674 · see citing works

Mutations in RPE65 cause autosomal recessive childhood–onset severe retinal dystrophy

Gu, S.-M., Thompson, D. A., Srikumari, C. R. S., Lorenz, B., Finckh, U., Nicoletti, A., Murthy, K. R., Rathmann, M., Kumaramanickavel, G., Denton, M. J., et al. (1997) · Nature Genetics · doi:10.1038/ng1097-194 · Cited by 663 · see citing works

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations: Safety and Efficacy in 15 Children and Adults Followed Up to 3 Years

Jacobson, S. G. (2012) · Archives of Ophthalmology · doi:10.1001/archophthalmol.2011.298 · Cited by 639 · see citing works

Genes and mutations causing retinitis pigmentosa

Daiger, S. P., Sullivan, L. S., & Bowne, S. J. (2013) · Clinical Genetics · doi:10.1111/cge.12203 · Cited by 625 · see citing works

Differential Uptake of Functionalized Polystyrene Nanoparticles by Human Macrophages and a Monocytic Cell Line

Lunov, O., Syrovets, T., Loos, C., Beil, J., Delacher, M., Tron, K., Nienhaus, G. U., Musyanovych, A., Mailänder, V., Landfester, K., et al. (2011) · ACS Nano · doi:10.1021/nn2000756 · Cited by 599 · see citing works

Preliminary biological evaluation of polyamidoamine (PAMAM) StarburstTM dendrimers

Roberts, J. C., Bhalgat, M. K., & Zera, R. T. (1996) · Journal of Biomedical Materials Research · doi:10.1002/(sici)1097-4636(199601)30:1 · Cited by 581 · see citing works

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

Simonelli, F., Maguire, A. M., Testa, F., Pierce, E. A., Mingozzi, F., Bennicelli, J. L., Rossi, S., Marshall, K. A., Banfi, S., Surace, E. M., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.277 · Cited by 553 · see citing works

A Powerful Nonviral Vector for In Vivo Gene Transfer into the Adult Mammalian Brain: Polyethylenimine

Abdallah, B., Hassan, A., Benoist, C., Goula, D., Behr, J. P., & DEMENEIX, B. A. (1996) · Human Gene Therapy · doi:10.1089/hum.1996.7.16-1947 · Cited by 541 · see citing works

Preparation and characterization of cationic PLGA nanospheres as DNA carriers

Kumar, M. N. V. R., Bakowsky, U., & Lehr, C. (2003) · Biomaterials · doi:10.1016/j.biomaterials.2003.08.069 · Cited by 493 · see citing works

Endocytosis and targeting of exogenous HIV‐1 Tat protein.

Mann, D. A., & Frankel, A. D. (1991) · The EMBO Journal · doi:10.1002/j.1460-2075.1991.tb07697.x · Cited by 485 · see citing works

Long-Term Restoration of Rod and Cone Vision by Single Dose rAAV-Mediated Gene Transfer to the Retina in a Canine Model of Childhood Blindness

Acland, G. M., Aguirre, G. K., Bennett, J., Alemán, T. S., Cideciyan, A. V., Bennicelli, J. L., Dejneka, N. S., Pearce-Kelling, S. E., Maguire, A. M., Palczewski, K., et al. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.08.008 · Cited by 478 · see citing works

Repression of genes by DNA methylation depends on CpG density and promoter strength: evidence for involvement of a methyl‐CpG binding protein.

Boyes, J., & Bird, A. (1992) · The EMBO Journal · doi:10.1002/j.1460-2075.1992.tb05055.x · Cited by 471 · see citing works

Rab GTPases, intracellular traffic and disease

Seabra, M. C., Mules, E. H., & Hume, A. N. (2002) · Trends in Molecular Medicine · doi:10.1016/s1471-4914(01)02227-4 · Cited by 468 · see citing works

Poly(ethylene glycol) on the liposome surface: on the mechanism of polymer-coated liposome longevity

Torchilin, V. P., Omelyanenko, V. G., Papisov, M. I., Bogdanov, A. A., Jr., Trubetskoy, V. S., Herron, J. N., & Gentry, C. A. (1994) · Biochimica et Biophysica Acta (BBA) - Biomembranes · doi:10.1016/0005-2736(94)90003-5 · Cited by 466 · see citing works

Protamine sulfate enhances lipid-mediated gene transfer

Sorgi, F., Bhattacharya, S. S., & Huang, L. (1997) · Gene Therapy · doi:10.1038/sj.gt.3300484 · Cited by 453 · see citing works

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

Cideciyan, A. V., Jacobson, S. G., Beltran, W. A., Sumaroka, A., Świder, M., Iwabe, S., Román, A. J., Olivares, M. B., Schwartz, S., Komáromy, A. M., et al. (2013) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1218933110 · Cited by 441 · see citing works

A quantitative analysis of intron effects on mammalian gene expression

Nott, A., MEISLIN, S. H., & Moore, M. J. (2003) · RNA · doi:10.1261/rna.5250403 · Cited by 417 · see citing works

Introduction of liposome-encapsulated SV40 DNA into cells.

Fraley, R. T., Subramani, S. E., Berg, P. R., & Papahadjopoulos, D. (1980) · Journal of Biological Chemistry · doi:10.1016/s0021-9258(19)70482-7 · Cited by 416 · see citing works

Overexpression of the epidermal growth factor receptor and its ligand transforming growth factor alpha is frequent in resectable non-small cell lung cancer but does not predict tumor progression.

Rusch, V. W., Klimstra, D. S., Venkatraman, E. S., Pisters, P. W. T., Langenfeld, J. E., & Dmitrovsky, E. (1997) · PubMed · Cited by 401 · see citing works

Loss of transcriptional activity of a transgene is accompanied by DNA methylation and histone deacetylation and is prevented by insulatorsPreprint

Pikaart, M. J., Targa, F. R., & Felsenfeld, G. (1998) · Genes & Development · doi:10.1101/gad.12.18.2852 · Cited by 398 · see citing works

Development of a novel systemic gene delivery system for cancer therapy with a tumor-specific cleavable PEG-lipid

Hatakeyama, H., Akita, H., Kogure, K., Oishi, M., Nagasaki, Y., Kihira, Y., Ueno, M., Kobayashi, H., Kikuchi, H., & Harashima, H. (2006) · Gene Therapy · doi:10.1038/sj.gt.3302843 · Cited by 397 · see citing works

Accelerated Discovery of Synthetic Transfection Vectors: Parallel Synthesis and Screening of a Degradable Polymer Library

Lynn, D. M., Anderson, D. G., Putnam, D. A., & Langer, R. (2001) · Journal of the American Chemical Society · doi:10.1021/ja016288p · Cited by 395 · see citing works

α-Thalassaemia caused by a polyadenylation signal mutation

Higgs, D. R., Goodbourn, S. E. Y., Lamb, J., Clegg, J. B., Weatherall, D. J., & Proudfoot, N. J. (1983) · Nature · doi:10.1038/306398a0 · Cited by 386 · see citing works

Improvement and Decline in Vision with Gene Therapy in Childhood Blindness

Jacobson, S. G., Cideciyan, A. V., Román, A. J., Sumaroka, A., Schwartz, S., Héon, E., & Hauswirth, W. W. (2015) · New England Journal of Medicine · doi:10.1056/nejmoa1412965 · Cited by 370 · see citing works

The role of PEG on the stability in digestive fluids and in vivo fate of PEG-PLA nanoparticles following oral administration

Tobío, M. T. B., Sánchez, A., Vila, A., Soriano, I., Évora, C., Vila-Jato, J. L., & Alonso, M. J. (2000) · Colloids and Surfaces B Biointerfaces · doi:10.1016/s0927-7765(99)00157-5 · Cited by 367 · see citing works

Transcriptional silencing is associated with extensive methylation of the CMV promoter following adenoviral gene delivery to muscle

Brooks, A. R., Harkins, R. N., Wang, P., Qian, H. S., Liu, P., & Rubanyi, G. M. (2004) · The Journal of Gene Medicine · doi:10.1002/jgm.516 · Cited by 363 · see citing works

Gene therapy with brain-derived neurotrophic factor as a protection: Retinal ganglion cells in a rat glaucoma model

Martin, K. R. G., Quigley, H. A., Zack, D. J., Levkovitch-Verbin, H., Kielczewski, J., Valenta, D., Baumrind, L., Pease, M. E., Klein, R. L., & Hauswirth, W. W. (2003) · Investigative Opthalmology & Visual Science · doi:10.1167/iovs.02-1332 · Cited by 362 · see citing works

Ocular Drug Delivery Targeting the Retina and Retinal Pigment Epithelium Using Polylactide Nanoparticles

Bourges, J., Gautier, S., Delié, F., Bejjani, R. A., Jeanny, J., Gurny, R., BenEzra, D., & Béhar‐Cohen, F. (2003) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.02-1068 · Cited by 357 · see citing works

Dynamic changes in the characteristics of cationic lipidic vectors after exposure to mouse serum: implications for intravenous lipofection

Li, S., Tseng, W.-C., Stolz, D. B., Wu, S.-Y., Watkins, S. C., & Huang, L. (1999) · Gene Therapy · doi:10.1038/sj.gt.3300865 · Cited by 351 · see citing works

Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element

Donello, J. E., Loeb, J. E., & Hope, T. J. (1998) · Journal of Virology · doi:10.1128/jvi.72.6.5085-5092.1998 · Cited by 350 · see citing works

Usher syndrome: Hearing loss, retinal degeneration and associated abnormalities

Mathur, P. D., & Yang, J. (2014) · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease · doi:10.1016/j.bbadis.2014.11.020 · Cited by 334 · see citing works

Nanoparticle diffusion in, and microrheology of, the bovine vitreous ex vivo

Xu, Q., Boylan, N. J., Suk, J. S., Wang, Y.-Y., Nance, E. A., Yang, J., McDonnell, P. J., Cone, R. A., Duh, E. J., & Hanes, J. S. (2013) · Journal of Controlled Release · doi:10.1016/j.jconrel.2013.01.018 · Cited by 332 · see citing works

Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2

Testa, F., Maguire, A. M., Rossi, S., Pierce, E. A., Melillo, P., Marshall, K. A., Banfi, S., Surace, E. M., Sun, J., Acerra, C., et al. (2013) · Ophthalmology · doi:10.1016/j.ophtha.2012.11.048 · Cited by 331 · see citing works

A robust system for production of minicircle DNA vectors

Kay, M. A., He, C.-Y., & Chen, Z.-Y. (2010) · Nature Biotechnology · doi:10.1038/nbt.1708 · Cited by 329 · see citing works

Macrophage Uptake of Core−Shell Nanoparticles Surface Modified with Poly(ethylene glycol)

Zahr, A. S., Davis, C. A., & Pishko, M. V. (2006) · Langmuir · doi:10.1021/la060951b · Cited by 324 · see citing works

In vitro polyadenylation is stimulated by the presence of an upstream intron.Preprint

Niwa, M., Rose, S. D., & Berget, S. M. (1990) · Genes & Development · doi:10.1101/gad.4.9.1552 · Cited by 314 · see citing works

Poly(lactic acid)-poly(ethylene glycol) nanoparticles as new carriers for the delivery of plasmid DNA

Perez, C., Sanchez, A., Putnam, D., Ting, D., Langer, R., & Alonso, M. J. (2001) · Journal of Controlled Release · doi:10.1016/s0168-3659(01)00397-2 · Cited by 313 · see citing works

CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression

Hyde, S. C., Pringle, I. A., Abdullah, S., Lawton, A. E., Davies, L. A., Varathalingam, A., Nunez-Alonso, G. A., Green, A.-M., Bazzani, R. P., Jones, S., et al. (2008) · Nature Biotechnology · doi:10.1038/nbt1399 · Cited by 308 · see citing works

Intervening sequences increase efficiency of RNA 3' processing and accumulation of cytoplasmic RNA

Huang, M. T. F., & Gorman, C. M. (1990) · Nucleic Acids Research · doi:10.1093/nar/18.4.937 · Cited by 303 · see citing works

Long circulating chitosan/PEG blended PLGA nanoparticle for tumor drug delivery

Parveen, S., & Sahoo, S. K. (2011) · European Journal of Pharmacology · doi:10.1016/j.ejphar.2011.09.023 · Cited by 302 · see citing works

Compacted DNA Nanoparticles Administered to the Nasal Mucosa of Cystic Fibrosis Subjects Are Safe and Demonstrate Partial to Complete Cystic Fibrosis Transmembrane Regulator Reconstitution

Konstan, M. W., Davis, P. B., Wagener, J. S., Hilliard, K. A., Stern, R. C., Milgram, L. J. H., Kowalczyk, T. H., Hyatt, S. L., Fink, T. L., Gedeon, C. R., et al. (2004) · Human Gene Therapy · doi:10.1089/hum.2004.15.1255 · Cited by 298 · see citing works

Effect of intron A from human cytomegalovirus (Towne) immediate-early gene on heterologous expression in mammalian cells

Chapman, B. S., Thayer, R. M., Vincent, K. A., et al. (1991) · Nucleic Acids Research · doi:10.1093/nar/19.14.3979 · Cited by 295 · see citing works

Novel Hyaluronic Acid-Chitosan Nanoparticles for Ocular Gene Therapy

de la Fuente, M., Seijo, B. A., & Alonso, M. J. (2008) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.07-1077 · Cited by 288 · see citing works

Import of Plasmid DNA into the Nucleus Is Sequence Specific

Dean, D. A. (1997) · Experimental Cell Research · doi:10.1006/excr.1996.3427 · Cited by 284 · see citing works

Monocyte chemoattractant protein 1 mediates retinal detachment-induced photoreceptor apoptosis

Nakazawa, T., Hisatomi, T., Nakazawa, C., Noda, K., Maruyama, K., She, H., Matsubara, A., Miyahara, S., Nakao, S., Yin, Y., et al. (2007) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0608167104 · Cited by 272 · see citing works

PARTICIPATION OF THE RETINAL PIGMENT EPITHELIUM IN THE ROD OUTER SEGMENT RENEWAL PROCESS

Young, R. W., & Bok, D. (1969) · Journal of Cell Biology · doi:10.1083/jcb.42.2.392 · Cited by 271 · see citing works

Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer

Bennicelli, J., Wright, J. F., Komaromy, A., Jacobs, J. B., Hauck, B., Zelenaia, O., Mingozzi, F., Hui, D., Chung, D., Rex, T. S., et al. (2008) · Molecular Therapy · doi:10.1038/sj.mt.6300389 · Cited by 267 · see citing works

A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis

Gill, D. R., Southern, K. W., Mofford, K., Seddon, T., Huang, L., Sorgi, F., Thomson, A. H., MacVinish, L. J., Ratcliff, R. A., Bilton, D., et al. (1997) · Gene Therapy · doi:10.1038/sj.gt.3300391 · Cited by 262 · see citing works

The use of biotinylated-EGF-modified gelatin nanoparticle carrier to enhance cisplatin accumulation in cancerous lungs via inhalation

Tseng, C., Su, W., Yen, K.-C., Yang, K., & Lin, F. (2009) · Biomaterials · doi:10.1016/j.biomaterials.2009.03.010 · Cited by 260 · see citing works

Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog

Narfstro¨m, K., Katz, M. L., Bragadottir, R., Seeliger, M., Boulanger, A., Redmond, T. M., Caro, L., Lai, C.-M., & Rakoczy, P. E. (2003) · Investigative Opthalmology & Visual Science · doi:10.1167/iovs.02-0595 · Cited by 252 · see citing works

Phase I Study of a Systemically Delivered p53 Nanoparticle in Advanced Solid Tumors

Senzer, N. N., Nemunaitis, J. J., Nemunaitis, D., Bedell, C., Edelman, G. M., Barve, M. A., Nunan, R., Pirollo, K. F., Rait, A. S., & Chang, E. H. (2013) · Molecular Therapy · doi:10.1038/mt.2013.32 · Cited by 250 · see citing works

A new DNA vehicle for nonviral gene delivery: supercoiled minicircle

Darquet, A.-M., Cameron, B., Wils, P., Scherman, D., & Crouzet, J. (1997) · Gene Therapy · doi:10.1038/sj.gt.3300540 · Cited by 250 · see citing works

Mannose Polyethylenimine Conjugates for Targeted DNA Delivery into Dendritic Cells

Diebold, S. S., Kursa, M., Wagner, E., Cotten, M. L., & Zenke, M. (1999) · Journal of Biological Chemistry · doi:10.1074/jbc.274.27.19087 · Cited by 248 · see citing works

Transfection efficiency and toxicity of polyethylenimine in differentiated Calu-3 and nondifferentiated COS-1 cell cultures

Florea, B. I., Meaney, C., Junginger, H. E., & Borchard, G. (2002) · AAPS PharmSci · doi:10.1208/ps040312 · Cited by 243 · see citing works

Sequence Requirements for Plasmid Nuclear Import

Dean, D. A., Dean, B. S., Muller, S., & Smith, L. C. (1999) · Experimental Cell Research · doi:10.1006/excr.1999.4716 · Cited by 235 · see citing works

Studies on the permeability of the blood-retinal barrier

Shakib, M., & Cunha-Vaz, J. G. (1966) · Experimental Eye Research · doi:10.1016/s0014-4835(66)80011-8 · Cited by 232 · see citing works

alpha 2 Macroglobulin binding to the plasma membrane of cultured fibroblasts. Diffuse binding followed by clustering in coated regions.

Willingham, M. C., Maxfield, F. R., & Pastan, I. H. (1979) · The Journal of Cell Biology · doi:10.1083/jcb.82.3.614 · Cited by 230 · see citing works

Congenital stationary night blindness in the dog: common mutation in the RPE65 gene indicates founder effect.

Aguirre, G. K., Baldwin, V. J., Pearce‐Kelling, S., Narfström, K. L., Ray, K., & Acland, G. M. (1998) · PubMed · Cited by 219 · see citing works

Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy

Kong, J., Kim, S.-R., Binley, K. E., Pata, I., Doi, K., Männik, J., Zernant-Rajang, J., Kan, O., Iqball, S., Naylor, S., et al. (2008) · Gene Therapy · doi:10.1038/gt.2008.78 · Cited by 216 · see citing works

Size-dependent disposition of nanoparticles and microparticles following subconjunctival administration

Amrite, A. C., & Kompella, U. B. (2005) · Journal of Pharmacy and Pharmacology · doi:10.1211/jpp.57.12.0005 · Cited by 213 · see citing works

A vector based on the SV40 origin of replication and chromosomal S/MARs replicates episomally in CHO cells

Piechaczek, C., Fetzer, C., Baiker, A. E., Bode, J., & Lipps, H. J. (1999) · Nucleic Acids Research · doi:10.1093/nar/27.2.426 · Cited by 211 · see citing works

Intramuscular Administration of AAV1-Lipoprotein Lipase ^S447X Lowers Triglycerides in Lipoprotein Lipase–Deficient Patients

Stroes, E. S., Nierman, M. C., Meulenberg, J. J., Franssen, R., Twisk, J., Henny, C. P., Maas, M. M., Zwinderman, A. H., Ross, C., Aronica, E., et al. (2008) · Arteriosclerosis, Thrombosis, and Vascular Biology · doi:10.1161/atvbaha.108.175620 · Cited by 209 · see citing works

Visualization of in vitro protein-rejecting properties of PEGylated stealth® polycyanoacrylate nanoparticles

Peracchia, M. T., Harnisch, S., Pinto‐Alphandary, H., Gulik, A., Dedieu, J., Desmaële, D., d’Angelo, J., Müller, R. H., & Couvreur, P. (1999) · Biomaterials · doi:10.1016/s0142-9612(99)00021-6 · Cited by 208 · see citing works

Efficient Non-Viral Ocular Gene Transfer with Compacted DNA Nanoparticles

Farjo, R., Skaggs, J., Quiambao, A. B., Cooper, M. J., & Naash, M. I. (2006) · PLoS ONE · doi:10.1371/journal.pone.0000038 · Cited by 208 · see citing works

Retinal degeneration 12 (rd12): a new, spontaneously arising mouse model for human Leber congenital amaurosis (LCA).

Pang, J., Chang, B., Hawes, N. L., Hurd, R. E., Davisson, M. T., Li, J., Noorwez, S. M., Malhotra, R., McDowell, J. H., Kaushal, S., et al. (2005) · PubMed · Cited by 206 · see citing works

PAMAM-PEG-PAMAM: Novel Triblock Copolymer as a Biocompatible and Efficient Gene Delivery Carrier

Kim, T., Seo, H. J., Choi, J. S., et al. (2004) · Biomacromolecules · doi:10.1021/bm049563j · Cited by 204 · see citing works

Optimization of AAV expression cassettes to improve packaging capacity and transgene expression in neurons

Choi, J.-H., Yu, N.-K., Baek, G.-C., Bakes, J., Seo, D., Nam, H. J., Baek, S. H., Lim, C.-S., Lee, Y.-S., & Kaang, B.-K. (2014) · Molecular Brain · doi:10.1186/1756-6606-7-17 · Cited by 197 · see citing works

Bioadhesive hyaluronan–chitosan nanoparticles can transport genes across the ocular mucosa and transfect ocular tissue

de la Fuente, M., Seijo, B., & Alonso, M. J. (2008) · Gene Therapy · doi:10.1038/gt.2008.16 · Cited by 194 · see citing works

Nanoparticles of Compacted DNA Transfect Postmitotic Cells

Liu, G., Li, D., Pasumarthy, M. K., Kowalczyk, T. H., Gedeon, C. R., Hyatt, S. L., Payne, J. M., Miller, T. J., Brunovskis, P., Fink, T. L., et al. (2003) · Journal of Biological Chemistry · doi:10.1074/jbc.m305776200 · Cited by 190 · see citing works

Biodegradable Polyester, Poly[α-(4-Aminobutyl)-l-Glycolic Acid], as a Non-Toxic Gene Carrier

Lim, Y., Han, S., Kong, H.-U., Lee, Y., Park, J. S., Jeong, B., & Kim, S. W. (2000) · Pharmaceutical Research · doi:10.1023/a:1007552007765 · Cited by 189 · see citing works

Vitreous: A Barrier to Nonviral Ocular Gene Therapy

Peeters, L., Sanders, N. N., Braeckmans, K., Boussery, K., Van de Voorde, J., De Smedt, S. C., & Demeester, J. (2005) · Investigative Opthalmology & Visual Science · doi:10.1167/iovs.05-0165 · Cited by 189 · cited 1× · see citing works

Influence of promoter and WHV post-transcriptional regulatory element on AAV-mediated transgene expression in the rat brain

Paterna, J.-C., Moccetti, T., Mura, A., Feldon, J., & Büeler, H. (2000) · Gene Therapy · doi:10.1038/sj.gt.3301221 · Cited by 189 · see citing works

Glial Cell Line Derived Neurotrophic Factor Delays Photoreceptor Degeneration in a Transgenic Rat Model of Retinitis Pigmentosa

Sanftner, L. H. M., Abel, H., Hauswirth, W. W., & Flannery, J. G. (2001) · Molecular Therapy · doi:10.1006/mthe.2001.0498 · Cited by 187 · see citing works

Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential.

(2008) · PubMed Central · Cited by 183 · see citing works

Genetic medicines: treatment strategies for hereditary disorders

O’Connor, T. P., & Crystal, R. G. (2006) · Nature Reviews Genetics · doi:10.1038/nrg1829 · Cited by 180 · see citing works

Nuclear Import of Plasmid DNA in Digitonin-permeabilized Cells Requires Both Cytoplasmic Factors and Specific DNA Sequences

Wilson, G. L., Dean, B. S., Wang, G., & Dean, D. A. (1999) · Journal of Biological Chemistry · doi:10.1074/jbc.274.31.22025 · Cited by 176 · see citing works

Folate-targeted nanoparticle delivery of chemo- and radiotherapeutics for the treatment of ovarian cancer peritoneal metastasis

Werner, M. E., Karve, S., Sukumar, R., Cummings, N. D., Copp, J. A., Chen, R. C., Zhang, T., & Wang, A. Z. (2011) · Biomaterials · doi:10.1016/j.biomaterials.2011.07.067 · Cited by 176 · see citing works

Scaffold-attached regions from the human interferon .beta. domain can be used to enhance the stable expression of genes under the control of various promoters

Klehr, D., Maass, K., & Bode, J. (1991) · Biochemistry · doi:10.1021/bi00219a015 · Cited by 175 · see citing works

Endocytic Crosstalk: Cavins, Caveolins, and Caveolae Regulate Clathrin-Independent Endocytosis

Chaudhary, N., Gomez, G. A., Howes, M. T., Lo, H. P., McMahon, K., Rae, J. A., Schieber, N. L., Hill, M. M. C., Gaus, K., Yap, A. S., et al. (2014) · PLoS Biology · doi:10.1371/journal.pbio.1001832 · Cited by 175 · see citing works

Investigating the movement of intravitreal human serum albumin nanoparticles in the vitreous and retina

Kim, H., Robinson, S. B., & Csaky, K. G. (2009) · Pharmaceutical Research · doi:10.1007/s11095-008-9745-6 · Cited by 174 · see citing works

Safety and Short-Term Toxicity of a Novel Cationic Lipid Formulation for Human Gene Therapy

San, H., Yang, Z., Pompili, V. J., et al. (1993) · Human Gene Therapy · doi:10.1089/hum.1993.4.6-781 · Cited by 174 · see citing works

Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes

Xu, R., Janson, C., Mastakov, M., Lawlor, P., Young, D., Mouravlev, A., Fitzsimons, H., Choi, K.-L., Ma, H., Dragunow, M., et al. (2001) · Gene Therapy · doi:10.1038/sj.gt.3301529 · Cited by 174 · see citing works

Assessment of Adenoviral Vector Safety and Toxicity: Report of the National Institutes of Health Recombinant DNA Advisory Committee

(2002) · Human Gene Therapy · doi:10.1089/10430340152712629 · Cited by 174 · see citing works

Antibody neutralization poses a barrier to intravitreal adeno-associated viral vector gene delivery to non-human primates

Kotterman, M. A., Yin, L., Strazzeri, J. M., Flannery, J. G., Merigan, W. H., & Schaffer, D. V. (2015) · Gene Therapy · doi:10.1038/gt.2014.115 · Cited by 169 · see citing works

The Role of Topical Antibiotic Prophylaxis to Prevent Endophthalmitis after Intravitreal Injection

Storey, P. P., Dollin, M. L., Pitcher, J. D., et al. (2013) · Ophthalmology · doi:10.1016/j.ophtha.2013.08.037 · Cited by 168 · see citing works

Relocalization of Apoptosis-Inducing Factor in Photoreceptor Apoptosis Induced by Retinal Detachment in Vivo

Hisatomi, T., Sakamoto, T., Murata, T., et al. (2001) · American Journal Of Pathology · doi:10.1016/s0002-9440(10)64078-3 · Cited by 168 · see citing works

Evidence for the Failure of Adeno-associated Virus Serotype 5 to Package a Viral Genome ≥8.2 kb

Lai, Y., Yue, Y., & Duan, D. (2010) · Molecular Therapy · doi:10.1038/mt.2009.256 · Cited by 166 · see citing works

Vitamin A and the role of the pigment epithelium during bleaching and regeneration of rhodopsin in the frog eye

Bridges, C. D. B. (1976) · Experimental Eye Research · doi:10.1016/0014-4835(76)90182-2 · Cited by 165 · see citing works

Nanoparticles for gene delivery to retinal pigment epithelial cells.

Bejjani, R. A., BenEzra, D., Cohen, H., Rieger, J., Andrieu‐Soler, C., Jeanny, J., Gollomb, G., & Béhar‐Cohen, F. (2005) · PubMed · Cited by 163 · see citing works

Enhanced transfection of tumor cellsin vivousing “Smart” pH-sensitive TAT-modified pegylated liposomes

Kale, A. A., & Torchilin, V. P. (2007) · Journal of drug targeting · doi:10.1080/10611860701498203 · Cited by 159 · see citing works

Coating nanocarriers with hyaluronic acid facilitates intravitreal drug delivery for retinal gene therapy

Martens, T., Remaut, K., Deschout, H. G., Engbersen, J. F. J., Hennink, W. E., van Steenbergen, M. J., Demeester, J., Smedt, S. C. D., & Braeckmans, K. (2015) · Journal of Controlled Release · doi:10.1016/j.jconrel.2015.01.030 · Cited by 157 · see citing works

Constitutive Activation of Photoreceptor Guanylate Cyclase by Y99C Mutant of GCAP-1

Dizhoor, A. M., Boikov, S. G., & Olshevskaya, E. V. (1998) · Journal of Biological Chemistry · doi:10.1074/jbc.273.28.17311 · Cited by 155 · see citing works

Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose

Wu, Z., Sun, J., Zhang, T., Yin, C., Yin, F., Van Dyke, T., Samulski, R. J., & Monahan, P. E. (2008) · Molecular Therapy · doi:10.1038/sj.mt.6300355 · Cited by 153 · see citing works

Intron-Mediated Enhancement of Gene Expression Independent of Unique Intron Sequences and Splicing

Rose, A. B., & Beliakoff, J. A. (2000) · PLANT PHYSIOLOGY · doi:10.1104/pp.122.2.535 · Cited by 153 · see citing works

HnRNP L binds a cis-acting RNA sequence element that enables intron-dependent gene expression.Preprint

Liu, X., & Mertz, J. E. (1995) · Genes & Development · doi:10.1101/gad.9.14.1766 · Cited by 151 · see citing works

DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice

Han, Z., Conley, S. M., Makkia, R. S., Cooper, M. J., & Naash, M. I. (2012) · Journal of Clinical Investigation · doi:10.1172/jci64833 · Cited by 150 · see citing works

Interference of poly(ethylene glycol)–lipid analogues with cationic-lipid-mediated delivery of oligonucleotides; role of lipid exchangeability and non-lamellar transitions

Shi, F., Wasungu, L., Nomden, A., Stuart, M. C. A., Polushkin, E., Engberts, J. B. F. N., & Hoekstra, D. (2002) · Biochemical Journal · doi:10.1042/bj20020590 · Cited by 149 · see citing works

Histone Modifications are Associated with the Persistence or Silencing of Vector-mediated Transgene Expression In Vivo

Riu, E., Chen, Z.-Y., Xu, H., He, C.-Y., & Kay, M. A. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300177 · Cited by 148 · see citing works

Enhanced drug targeting by attachment of an anti αv integrin antibody to doxorubicin loaded human serum albumin nanoparticles

Wagner, S., Rothweiler, F., Anhorn, M. G., Sauer, D., Riemann, I., Weiss, E. C., Katsen‐Globa, A., Michaelis, M., Cinatl, J. J., Schwartz, D., et al. (2009) · Biomaterials · doi:10.1016/j.biomaterials.2009.11.093 · Cited by 147 · see citing works

Efficient gene silencing in metastatic tumor by siRNA formulated in surface-modified nanoparticles

Li, S., Chono, S., & Huang, L. (2007) · Journal of Controlled Release · doi:10.1016/j.jconrel.2007.11.002 · Cited by 147 · see citing works

Pegylation of liposomes favours the endosomal degradation of the delivered phosphodiester oligonucleotides

Remaut, K., Lucas, B., Braeckmans, K., Demeester, J., & Smedt, S. C. D. (2006) · Journal of Controlled Release · doi:10.1016/j.jconrel.2006.10.029 · Cited by 146 · see citing works

Plasmid size up to 20 kbp does not limit effective in vivo lung gene transfer using compacted DNA nanoparticles

Fink, T. L., Klepcyk, P. J., Oette, S. M., Gedeon, C. R., Hyatt, S. L., Kowalczyk, T. H., Moen, R. C., & Cooper, M. J. (2006) · Gene Therapy · doi:10.1038/sj.gt.3302761 · Cited by 139 · see citing works

Cationic Hyperbranched Poly(amino ester): A Novel Class of DNA Condensing Molecule with Cationic Surface, Biodegradable Three-Dimensional Structure, and Tertiary Amine Groups in the Interior

Lim, Y.-B., Kim, S.-M., Lee, Y., Lee, W.-K., Yang, T.-G., Lee, M.-J., Suh, H., & Park, J.-S. (2001) · Journal of the American Chemical Society · doi:10.1021/ja005715g · Cited by 138 · see citing works

Virus-sized self-assembling lamellar complexes between plasmid DNA and cationic micelles promote gene transfer

Pitard, B., Aguerre, O., Airiau, M., et al. (1997) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.94.26.14412 · Cited by 134 · see citing works

Human serum albumin nanoparticles for efficient delivery of Cu, Zn superoxide dismutase gene.

Mo, Y., Barnett, M. E., Takemoto, D. J., Davidson, H. J., & Kompella, U. B. (2007) · PubMed Central · Cited by 133 · see citing works

Synthesis and characterization of folate-PEG-grafted-hyperbranched-PEI for tumor-targeted gene delivery

Liang, B., He, M., Xiao, Z., et al. (2008) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2008.01.024 · Cited by 133 · see citing works

Albumin Nanoparticles for the Intravitreal Delivery of Anticytomegaloviral Drugs

Irache, J. M., Merodio, M., Arnedo, A., Camapanero, M., Mirshahi, M., & Espuelas, S. (2005) · Mini-Reviews in Medicinal Chemistry · doi:10.2174/1389557053175335 · Cited by 131 · see citing works

Improved recombinant gene expression in CHO cells using matrix attachment regions

Kim, S., Kim, J.-S., Park, D.-H., Kang, H. S., Yoon, J., Baek, K., & Yoon, Y. (2003) · Journal of Biotechnology · doi:10.1016/j.jbiotec.2003.09.015 · Cited by 129 · see citing works

Review and update on the molecular basis of Leber congenital amaurosis

Chacon-Camacho, O. F. (2015) · World Journal of Clinical Cases · doi:10.12998/wjcc.v3.i2.112 · Cited by 128 · see citing works

CNOT3 Is a Modifier of PRPF31 Mutations in Retinitis Pigmentosa with Incomplete Penetrance

Venturini, G., Rose, A. M., Shah, A. Z., et al. (2012) · PLoS Genetics · doi:10.1371/journal.pgen.1003040 · Cited by 127 · see citing works

Effect of circulation on the disposition and ocular tissue distribution of 20 nm nanoparticles after periocular administration.

Amrite, A. C., Edelhauser, H. F., Singh, S. R., & Kompella, U. B. (2008) · PubMed Central · Cited by 125 · see citing works

Efficient Transfer of Genes into Murine Cardiac Grafts by Starburst Polyamidoamine Dendrimers

Qin, L., Pahud, D. R., Ding, Y., Bielinska, A. U., Kukowska‐Latallo, J. F., Baker, J. R., & Bromberg, J. S. (1998) · Human Gene Therapy · doi:10.1089/hum.1998.9.4-553 · Cited by 124 · see citing works

Surfactants, not size or zeta-potential influence blood–brain barrier passage of polymeric nanoparticles

Voigt, N., Henrich‐Noack, P., Kockentiedt, S., Hintz, W., Tomas, J., & Sabel, B. A. (2014) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2014.02.013 · Cited by 122 · see citing works

Incidence of rhegmatogenous retinal detachments after intravitreal antivascular endothelial factor injections

Meyer, C. H., Michels, S., Rodrigues, E. B., Hager, A., Mennel, S., Schmidt, J. C., Helb, H. –., & Farah, M. E. (2010) · Acta Ophthalmologica · doi:10.1111/j.1755-3768.2010.02064.x · Cited by 122 · see citing works

Gene delivery to mitotic and postmitotic photoreceptors Via compacted DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa

Cai, X., Conley, S. M., Nash, Z. A., Fliesler, S. J., Cooper, M. J., & Naash, M. I. (2009) · The FASEB Journal · doi:10.1096/fj.09-139147 · Cited by 122 · see citing works

Genomic discovery of potent chromatin insulators for human gene therapy

Liu, M., Maurano, M. T., Wang, H., Qi, H., Song, C.-Z., Navas, P. A., Emery, D. W., Stamatoyannopoulos, J. A., & Stamatoyannopoulos, G. (2015) · Nature Biotechnology · doi:10.1038/nbt.3062 · Cited by 121 · see citing works

Minicircle DNA Vectors Achieve Sustained Expression Reflected by Active Chromatin and Transcriptional Level

Gracey, L. E., Maniar, J. M., Chen, Z.-Y., Lu, J., Fire, A. Z., & Kay, M. A. (2012) · Molecular Therapy · doi:10.1038/mt.2012.244 · Cited by 121 · see citing works

The Effect of Peripherin/rds Haploinsufficiency on Rod and Cone Photoreceptors

Cheng, T., Peachey, N. S., Li, S., Goto, Y., Cao, Y., & Naash, M. I. (1997) · Journal of Neuroscience · doi:10.1523/jneurosci.17-21-08118.1997 · Cited by 119 · see citing works

Nanoparticle-Assisted Targeted Delivery of Eye-Specific Genes to Eyes Significantly Improves the Vision of Blind Mice In Vivo

Rajala, A., Wang, Y., Zhu, Y., Ranjo‐Bishop, M., Ma, J., Mao, C., & Rajala, R. V. S. (2014) · Nano Letters · doi:10.1021/nl502275s · Cited by 118 · see citing works

Cisplatin‐incorporated hyaluronic acid nanoparticles based on ion‐complex formation

Jeong, Y., Kim, S., Jin, S., et al. (2007) · Journal of Pharmaceutical Sciences · doi:10.1002/jps.21103 · Cited by 116 · see citing works

Transduction of Photoreceptors With Equine Infectious Anemia Virus Lentiviral Vectors: Safety and Biodistribution of StarGen for Stargardt Disease

Binley, K., Widdowson, P., Loader, J., Kelleher, M., Iqball, S., Ferrige, G., de Belin, J., Carlucci, M., Angell-Manning, D., Hurst, F., et al. (2013) · Investigative Opthalmology & Visual Science · doi:10.1167/iovs.13-11871 · Cited by 115 · see citing works

Hepatitis B virus RNA element that facilitates accumulation of surface gene transcripts in the cytoplasm

Huang, Z., & Yen, T. S. B. (1994) · Journal of Virology · doi:10.1128/jvi.68.5.3193-3199.1994 · Cited by 113 · see citing works

Optimization of Plasmid Vectors for High-Level Expression in Lung Epithelial Cells

Yew, N. S., Wysokenski, D. M., Wang, K. X., Ziegler, R. J., Marshall, J., McNeilly, D., Cherry, M., Osburn, W., & Cheng, S. H. (1997) · Human Gene Therapy · doi:10.1089/hum.1997.8.5-575 · Cited by 113 · see citing works

A Partial Structural and Functional Rescue of a Retinitis Pigmentosa Model with Compacted DNA Nanoparticles

Cai, X., Nash, Z., Conley, S. M., Fliesler, S. J., Cooper, M. J., & Naash, M. I. (2009) · PLoS ONE · doi:10.1371/journal.pone.0005290 · Cited by 109 · see citing works

A novel gene therapy vector based on hyaluronic acid and solid lipid nanoparticles for ocular diseases

Apaolaza, P. S., Delgado, D., del Pozo‐Rodríguez, A., Rodríguez‐Gascón, A., & Solinís, M. Á. (2014) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2014.02.038 · Cited by 108 · see citing works

Nanoparticle-based technologies for retinal gene therapy

Adijanto, J., & Naash, M. I. (2015) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2014.12.028 · Cited by 107 · see citing works

Condensation of plasmid DNA with polylysine improves liposome-mediated gene transfer into established and primary muscle cells.

Vitiello, L., Chonn, A., Wasserman, J. D., Duff, C., & Worton, R. G. (1996) · PubMed · Cited by 104 · see citing works

EIAV-Based Retinal Gene Therapy in the shaker1 Mouse Model for Usher Syndrome Type 1B: Development of UshStat

Zallocchi, M., Binley, K. M., Lad, Y., Ellis, S., Widdowson, P. S., Iqball, S., Scripps, V., Kelleher, M., Loader, J., Miskin, J., et al. (2014) · PLoS ONE · doi:10.1371/journal.pone.0094272 · Cited by 103 · see citing works

Opsonization, Biodistribution, Cellular Uptake and Apoptosis Study of PEGylated PBCA Nanoparticle as Potential Drug Delivery Carrier

Chaudhari, K. R., Ukawala, M., Manjappa, A. S., Kumar, A., Mundada, P. K., Mishra, A. K., Mathur, R., Mönkkönen, J., & Murthy, R. S. R. (2011) · Pharmaceutical Research · doi:10.1007/s11095-011-0510-x · Cited by 103 · see citing works

An episomally replicating vector binds to the nuclear matrix protein SAF‐A in vivo

Jenke, B. H. C., Fetzer, C. P., Stehle, I. M., Jönsson, F., Fackelmayer, F. O., Conradt, H. S., Bode, J., & Lipps, H. J. (2002) · EMBO Reports · doi:10.1093/embo-reports/kvf070 · Cited by 102 · see citing works

Evaluation of a novel biomaterial in the suprachoroidal space of the rabbit eye.

Einmahl, S., Savoldelli, M., D'Hermies, F., Tabatabay, C., Gurny, R., & Béhar‐Cohen, F. (2002) · PubMed · Cited by 102 · see citing works

Understanding the mechanism of protamine in solid lipid nanoparticle-based lipofection: The importance of the entry pathway

Delgado, D., del Pozo‐Rodríguez, A., Solinís, M. Á., & Rodríguez‐Gascón, A. (2011) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2011.06.005 · Cited by 101 · see citing works

Overcoming the polyethylene glycol dilemma via pathological environment-sensitive change of the surface property of nanoparticles for cellular entry

Hama, S., Itakura, S., Nakai, M., Nakayama, K., Morimoto, S., Suzuki, S., & Kogure, K. (2015) · Journal of Controlled Release · doi:10.1016/j.jconrel.2015.03.011 · Cited by 101 · see citing works

Evaluation of Cellular Uptake and Gene Transfer Efficiency of Pegylated Poly- l -lysine Compacted DNA: Implications for Cancer Gene Therapy

Walsh, M., Tangney, M., O’Neill, M. J., Larkin, J. O., Soden, D. M., McKenna, S. L., Darcy, R., O’Sullivan, G. C., & O’Driscoll, C. M. (2006) · Molecular Pharmaceutics · doi:10.1021/mp0600034 · Cited by 100 · see citing works

Extraction and characterization of the tissue forms of collagen types II and IX from bovine vitreous

Bishop, P. N., Crossman, M. V., McLeod, D., & Ayad, S. (1994) · Biochemical Journal · doi:10.1042/bj2990497 · Cited by 100 · see citing works

Preclinical Potency and Safety Studies of an AAV2-Mediated Gene Therapy Vector for the Treatment of MERTK Associated Retinitis Pigmentosa

Conlon, T. J., Deng, W., Erger, K. E., Cossette, T. L., Pang, J., Ryals, R. C., Clément, N., Cleaver, B. D., McDoom, I., Boye, S. L., et al. (2013) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2013.037 · Cited by 99 · see citing works

Allelic Heterogeneity and Genetic Modifier Loci Contribute to Clinical Variation in Males with X-Linked Retinitis Pigmentosa Due to RPGR Mutations

Fahim, A. T., Bowne, S. J., Sullivan, L. S., Webb-Jones, K., Williams, J. T., Wheaton, D. K., Birch, D. G., & Daiger, S. P. (2011) · PLoS ONE · doi:10.1371/journal.pone.0023021 · Cited by 98 · see citing works

Effect of a DNA nuclear targeting sequence on gene transfer and expression of plasmids in the intact vasculature

Young, J. L., Benoit, J. N., & Dean, D. A. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302021 · Cited by 97 · see citing works

Nanoparticle-Mediated Expression of an Angiogenic Inhibitor Ameliorates Ischemia-Induced Retinal Neovascularization and Diabetes-Induced Retinal Vascular Leakage

Park, K., Chen, Y., Hu, Y., Mayo, A. S., Kompella, U. B., Longeras, R., & Ma, J. (2009) · Diabetes · doi:10.2337/db08-1327 · Cited by 97 · see citing works

Trial End Points and Natural History in Patients With G11778A Leber Hereditary Optic Neuropathy: Preparation for Gene Therapy Clinical Trial

Lam, B. L., Feuer, W. J., Schiffman, J. C., Porciatti, V., Vandenbroucke, R., Rosa, P. R., Gregori, G., & Guy, J. (2014) · JAMA Ophthalmology · doi:10.1001/jamaophthalmol.2013.7971 · Cited by 96 · see citing works

Genetic and phenotypic heterogeneity in pattern dystrophy

Francis, P. J. (2005) · British Journal of Ophthalmology · doi:10.1136/bjo.2004.062695 · Cited by 96 · see citing works

Significant Inhibition of Corneal Scarring In Vivo with Tissue-Selective, Targeted AAV5 Decorin Gene Therapy

Mohan, R. R., Tandon, A., Sharma, A., Cowden, J. W., & Tovey, J. C. K. (2011) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.11-7357 · Cited by 94 · see citing works

Measuring The Intravitreal Mobility of Nanomedicines with Single-Particle Tracking Microscopy

Martens, T., Vercauteren, D., Forier, K., Deschout, H. G., Remaut, K., Paesen, R., Ameloot, M. M., Engbersen, J. F. J., Demeester, J., Smedt, S. C. D., et al. (2013) · Nanomedicine · doi:10.2217/nnm.12.202 · Cited by 94 · see citing works

Lipid Nanoparticles for Ocular Gene Delivery

Wang, Y., Rajala, A., & Rajala, R. (2015) · Journal of Functional Biomaterials · doi:10.3390/jfb6020379 · Cited by 93 · see citing works

Hyaluronan-modified core–shell liponanoparticles targeting CD44-positive retinal pigment epithelium cells via intravitreal injection

Gan, L., Wang, J., Zhao, Y., Chen, D., Zhu, C., Liu, J., & Gan, Y. (2013) · Biomaterials · doi:10.1016/j.biomaterials.2013.04.035 · Cited by 93 · see citing works

PEGylation of biodegradable dextran nanogels for siRNA delivery

Naeye, B., Raemdonck, K., Remaut, K., Sproat, B. S., Demeester, J., & Smedt, S. C. D. (2010) · European Journal of Pharmaceutical Sciences · doi:10.1016/j.ejps.2010.04.010 · Cited by 93 · see citing works

Minimal toxicity of stabilized compacted DNA nanoparticles in the murine lung

Ziady, A. G., Gedeon, C. R., Muhammad, O., Stillwell, V., Oette, S. M., Fink, T. L., Quan, W., Kowalczyk, T. H., Hyatt, S. L., Payne, J. M., et al. (2003) · Molecular Therapy · doi:10.1016/j.ymthe.2003.09.002 · Cited by 93 · see citing works

Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo

Tolmachova, T., Tolmachov, O. E., Barnard, A. R., Silva, S. R. D., Lipinski, D. M., Walker, N. J., MacLaren, R. E., & Seabra, M. C. (2013) · Journal of Molecular Medicine · doi:10.1007/s00109-013-1006-4 · Cited by 92 · see citing works

The Use of Chromatin Insulators to Improve the Expression and Safety of Integrating Gene Transfer Vectors

Emery, D. W. (2011) · Human Gene Therapy · doi:10.1089/hum.2010.233 · Cited by 92 · see citing works

Dextran and Protamine-Based Solid Lipid Nanoparticles as Potential Vectors for the Treatment of X-Linked Juvenile Retinoschisis

Delgado, D., del Pozo‐Rodríguez, A., Solinís, M. Á., Avilés-Triqueros, M., Weber, B. H. F., Fernández, E., & Rodríguez‐Gascón, A. (2012) · Human Gene Therapy · doi:10.1089/hum.2011.115 · Cited by 92 · see citing works

Extended Core Sequences from the cHS4 Insulator Are Necessary for Protecting Retroviral Vectors from Silencing Position Effects

Aker, M., Tubb, J., Groth, A. C., Bukovsky, A. A., Bell, A. C., Felsenfeld, G., Kiem, H., Stamatoyannopoulos, G., & Emery, D. W. (2007) · Human Gene Therapy · doi:10.1089/hum.2007.021 · Cited by 92 · see citing works

Dextran–protamine–solid lipid nanoparticles as a non-viral vector for gene therapy: In vitro characterization and in vivo transfection after intravenous administration to mice

Delgado, D., Rodríguez‐Gascón, A., del Pozo‐Rodríguez, A., Echevarrı́a, E., de Garibay, A. P. R., Rodríguez, J. M., & Solinís, M. Á. (2012) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2011.12.052 · Cited by 91 · see citing works

Ultrapure chitosan oligomers as carriers for corneal gene transfer

Klausner, E. A., Zhang, Z., Chapman, R. L., Multack, R. F., & Volin, M. V. (2009) · Biomaterials · doi:10.1016/j.biomaterials.2009.10.031 · Cited by 88 · see citing works

Treatment of ocular disorders by gene therapy

Solinís, M. Á., del Pozo-Rodríguez, A., Apaolaza, P. S., & Rodríguez-Gascón, A. (2015) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2014.12.022 · Cited by 88 · see citing works

Chromosome boundary elements and regulation of heterochromatin spreading

Wang, J., Lawry, S. T., Cohen, A. L., & Jia, S. (2014) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-014-1725-x · Cited by 86 · see citing works

Comparative Analysis of DNA Nanoparticles and AAVs for Ocular Gene Delivery

Han, Z., Conley, S. M., Makkia, R. S., Guo, J., Cooper, M. J., & Naash, M. I. (2012) · PLoS ONE · doi:10.1371/journal.pone.0052189 · Cited by 84 · see citing works

Breaking down the wall: the nuclear envelope during mitosis

Smoyer, C. J., & Jaspersen, S. L. (2013) · Current Opinion in Cell Biology · doi:10.1016/j.ceb.2013.08.002 · Cited by 83 · see citing works

Stable and efficient intraocular gene transfer using pseudotyped EIAV lentiviral vectors

Balaggan, K. S., Binley, K. E., Esapa, M., Iqball, S., Askham, Z., Kan, O., Tschernutter, M., Bainbridge, J. W., Naylor, S., & Ali, R. R. (2005) · The Journal of Gene Medicine · doi:10.1002/jgm.845 · Cited by 83 · see citing works

Double Knockdown of Prolyl Hydroxylase and Factor-Inhibiting Hypoxia-Inducible Factor With Nonviral Minicircle Gene Therapy Enhances Stem Cell Mobilization and Angiogenesis After Myocardial Infarction

Huang, M., Nguyen, P. K. P., Jia, F., Hu, S., Gong, Y., de Almeida, P. E., Wang, L., Nag, D., Kay, M. A., Giaccia, A. J., et al. (2011) · Circulation · doi:10.1161/circulationaha.110.014019 · Cited by 82 · see citing works

Polyethyleneimine-mediated transfection of cultured postmitotic neurons from rat sympathetic ganglia and adult human retina

Horbinski, C., Stachowiak, M. K., Higgins, D. M., & Finnegan, S. G. (2001) · BMC Neuroscience · doi:10.1186/1471-2202-2-2 · Cited by 80 · see citing works

<i>RPE65</i>Gene Delivery Restores Isomerohydrolase Activity and Prevents Early Cone Loss in<i>Rpe65^−/−</i>Mice

Chen, Y., Moiseyev, G., Takahashi, Y., & Ma, J.-X. (2006) · Investigative Opthalmology &amp; Visual Science · doi:10.1167/iovs.05-0965 · Cited by 80 · see citing works

Long-Term Rescue of Retinal Structure and Function by Rhodopsin RNA Replacement with a Single Adeno-Associated Viral Vector in P23H RHO Transgenic Mice

Mao, H., Gorbatyuk, M. S., Rossmiller, B. P., Hauswirth, W. W., & Lewin, A. S. (2012) · Human Gene Therapy · doi:10.1089/hum.2011.213 · Cited by 79 · see citing works

Muscle-specific enhancement of gene expression by incorporation of SV40 enhancer in the expression plasmid

Li, S., MacLaughlin, F. C., Fewell, J. G., Gondo, M., Wang, J., Nicol, F., Dean, D. A., & Smith, L. C. (2001) · Gene Therapy · doi:10.1038/sj.gt.3301419 · Cited by 77 · see citing works

Induction of Endoplasmic Reticulum Stress Genes, BiP and Chop , in Genetic and Environmental Models of Retinal Degeneration

Kroeger, H., Messah, C., Ahern, K., Gee, J. M., Joseph, V. T., Matthes, M. T., Yasumura, D., Gorbatyuk, M. S., Chiang, W.-C. J., LaVail, M. M., et al. (2012) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.12-10221 · Cited by 77 · see citing works

Low generation PAMAM dendrimer and CpG free plasmids allow targeted and extended transgene expression in tumors after systemic delivery

Navarro, G., Maiwald, G., Haase, R., et al. (2010) · Journal of Controlled Release · doi:10.1016/j.jconrel.2010.04.030 · Cited by 76 · see citing works

S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA

Koirala, A., Makkia, R. S., Conley, S. M., Cooper, M. J., & Naash, M. I. (2013) · Human Molecular Genetics · doi:10.1093/hmg/ddt013 · Cited by 75 · see citing works

Antiangiogenic gene therapy with systemically administered sFlt-1 plasmid DNA in engineered gelatin-based nanovectors

Kommareddy, S., & Amiji, M. M. (2007) · Cancer Gene Therapy · doi:10.1038/sj.cgt.7701041 · Cited by 74 · see citing works

Mouse Choroideremia Gene Mutation Causes Photoreceptor Cell Degeneration and is not Transmitted through the Female Germline

van den Hurk, J. A. J. M., Hendriks, W. J. A. J., van de Pol, D. J. R., Oerlemans, F. T. J. J., Jaissle, G. B., Rüther, K., Köhler, K., Hartmann, J., Zrenner, E., van Bokhoven, H., et al. (1997) · Human Molecular Genetics · doi:10.1093/hmg/6.6.851 · Cited by 74 · see citing works

Hybrid Nanoparticle Design Based on Cationized Gelatin and the Polyanions Dextran Sulfate and Chondroitin Sulfate for Ocular Gene Therapy

Zorzi, G. K., Párraga, J. E., Seijo, B., & Sánchez, A. (2011) · Macromolecular Bioscience · doi:10.1002/mabi.201100005 · Cited by 73 · see citing works

Compacted DNA Nanoparticle Gene Transfer of GDNF to the Rat Striatum Enhances the Survival of Grafted Fetal Dopamine Neurons

Yurek, D. M., Fletcher‐Turner, A., Kowalczyk, T. H., Padegimas, L., & Cooper, M. J. (2009) · Cell Transplantation · doi:10.3727/096368909x12483162196881 · Cited by 73 · see citing works

Mutations in ARL2BP, Encoding ADP-Ribosylation-Factor-Like 2 Binding Protein, Cause Autosomal-Recessive Retinitis Pigmentosa

Davidson, A. E., Schwarz, N., Zelinger, L., Stern-Schneider, G., Shoemark, A., Spitzbarth, B., Gross, M., Laxer, U., Sosna, J., Sergouniotis, P. I., et al. (2013) · The American Journal of Human Genetics · doi:10.1016/j.ajhg.2013.06.003 · Cited by 73 · see citing works

Biodegradable nanoparticles composed of dendrigraft poly-l-lysine for gene delivery

Kodama, Y., Nakamura, T., Kurosaki, T., et al. (2014) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2014.04.013 · Cited by 73 · see citing works

In-depth mass spectrometric mapping of the human vitreous proteome

Aretz, S., Krohne, T. U., Kammerer, K., Warnken, U. H., Hotz‐Wagenblatt, A., Bergmann, M., Stanzel, B. V., Kempf, T., Holz, F. G., Schnölzer, M., et al. (2013) · Proteome Science · doi:10.1186/1477-5956-11-22 · Cited by 73 · see citing works

Cell penetrating peptide POD mediates delivery of recombinant proteins to retina, cornea and skin

Johnson, L. N., Cashman, S. M., Read, S. P., & Kumar‐Singh, R. (2009) · Vision Research · doi:10.1016/j.visres.2009.08.028 · Cited by 72 · see citing works

Ocular gene therapies in clinical practice: viral vectors and nonviral alternatives

Bordet, T., & Behar-Cohen, F. (2019) · Drug Discovery Today · doi:10.1016/j.drudis.2019.05.038 · Cited by 120 · see citing works

The Ocular Gene Delivery Landscape

Sahu, B., Chug, I., & Khanna, H. (2021) · Biomolecules · doi:10.3390/biom11081135 · Cited by 28 · see citing works

In vivo gene transfer of insulin gene into neonatal rats by the HVJ-liposome method resulted in sustained transgene expression.

Tomita, N., Morishita, R., Higaki, J., et al. (1996) · PubMed · Cited by 23 · see citing works

Advanced gene delivery : from concepts to pharmaceutical products

Rolland, A. P. (1999) · Cited by 25 · see citing works

Targeting of drugs 5 : strategies for oligonucleotide and gene delivery in therapy

Gregoriadis, G. C., & McCormack, B. (1996) · Plenum Press eBooks · Cited by 13 · see citing works

Microbubble-enhanced ultrasound for vascular gene delivery

Lawrie, A., Brisken, A. F., Francis, S., et al. (2000) · Gene Therapy · doi:10.1038/sj.gt.3301339 · Cited by 343 · see citing works

Bacteria in gene therapy: bactofection versus alternative gene therapy

Pálffy, R., Gardlík, R., Hodosy, J., Behuliak, M., Resko, P., Radvánský, J., & Celec, P. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302635 · Cited by 103 · see citing works