Comparative analysis of in vivo and in vitro AAV vector transduction in the neonatal mouse retina: Effects of serotype and site of administration

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

Gao, G.-P., Alvira, M. R., Wang, L., Calcedo, R., Johnston, J., & Wilson, J. M. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.182412299 · Cited by 1,520 · see citing works

Clades of Adeno-Associated Viruses Are Widely Disseminated in Human Tissues

Gao, G., Vandenberghe, L. H., Alvira, M. R., Lu, Y., Calcedo, R., Zhou, X., & Wilson, J. M. (2004) · Journal of Virology · doi:10.1128/jvi.78.12.6381-6388.2004 · Cited by 1,070 · see citing works

Cross-Packaging of a Single Adeno-Associated Virus (AAV) Type 2 Vector Genome into Multiple AAV Serotypes Enables Transduction with Broad Specificity

Rabinowitz, J. E., Rolling, F., Li, C., Conrath, H., Xiao, W., Xiao, X., & Samulski, R. J. (2002) · Journal of Virology · doi:10.1128/jvi.76.2.791-801.2002 · Cited by 762 · see citing works

From the Cover: Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system

Davidson, B. L., Stein, C. S., Heth, J., Martins, I., Kotin, R. M., Derksen, T. A., Zabner, J., Ghodsi, A., & Chiorini, J. A. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.050581197 · Cited by 679 · see citing works

Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8

Inagaki, K., Fuess, S., Storm, T. A., Gibson, G. A., Mctiernan, C. F., Kay, M. A., & Nakai, H. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.03.014 · Cited by 610 · see citing works

A "humanized" green fluorescent protein cDNA adapted for high-level expression in mammalian cells

Zolotukhin, S., Potter, M., Hauswirth, W. W., Guy, J., & Muzyczka, N. (1996) · Journal of Virology · doi:10.1128/jvi.70.7.4646-4654.1996 · Cited by 609 · see citing works

Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors

Zolotukhin, S., Potter, M., Zolotukhin, I., Sakai, Y., Loiler, S., Fraites, T. J., Chiodo, V. A., Philipsberg, T., Muzyczka, N., Hauswirth, W. W., et al. (2002) · Methods · doi:10.1016/s1046-2023(02)00220-7 · Cited by 556 · see citing works

Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system

Davidson, B. L., Stein, C. S., Heth, J. A., Martins, I., Kotin, R. M., Derksen, T. A., Zabner, J., Ghodsi, A., & Chiorini, J. A. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.97.7.3428 · Cited by 531 · 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

Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus

Flannery, J. G., Zolotukhin, S., Vaquero, M. I., LaVail, M. M., Muzyczka, N., & Hauswirth, W. W. (1997) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.94.13.6916 · Cited by 407 · see citing works

Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model

Auricchio, A. (2001) · Human Molecular Genetics · doi:10.1093/hmg/10.26.3075 · Cited by 394 · see citing works

AAV-mediated expression of CNTF promotes long-term survival and regeneration of adult rat retinal ganglion cells

Leaver, S. G., Cui, Q., Plant, G. W., Arulpragasam, A., Hisheh, S., Verhaagen, J., & Harvey, A. R. (2006) · Gene Therapy · doi:10.1038/sj.gt.3302791 · Cited by 270 · see citing works

Gene transfer into the mouse retina mediated by an adeno-associated viral vector

Ali, R. R. (1996) · Human Molecular Genetics · doi:10.1093/hmg/5.5.591 · Cited by 255 · see citing works

Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina

Bennett, J., Maguire, A. M., Cideciyan, A. V., Schnell, M. A., Glover, E., Anand, V., Alemán, T. S., Chirmule, N. B., Gupta, A. R., Huang, Y., et al. (1999) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.96.17.9920 · Cited by 242 · see citing works

Two novel adeno-associated viruses from cynomolgus monkey: pseudotyping characterization of capsid protein

Mori, S., Wang, L., Takeuchi, T., & Kanda, T. (2004) · Virology · doi:10.1016/j.virol.2004.10.012 · Cited by 240 · see citing works

Restoration of cone vision in a mouse model of achromatopsia

Alexander, J. J., Umino, Y., Everhart, D., Chang, B., Min, S.-H., Li, Q., Timmers, A. M., Hawes, N. L., Pang, J., Barlow, R. B., et al. (2007) · Nature Medicine · doi:10.1038/nm1596 · Cited by 218 · see citing works

Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery

Weber, M., Rabinowitz, J., Provost, N., Conrath, H., Folliot, S., Briot, D., Chérel, Y., Chenuaud, P., Samulski, J., Moullier, P., et al. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(03)00098-4 · Cited by 203 · see citing works

Virus-Mediated Transduction of Murine Retina with Adeno-Associated Virus: Effects of Viral Capsid and Genome Size

Yang, G. S., Schmidt, M., Yan, Z., Lindbloom, J. D., Harding, T. C., Donahue, B. A., Engelhardt, J. F., Kotin, R. M., & Davidson, B. L. (2002) · Journal of Virology · doi:10.1128/jvi.76.15.7651-7660.2002 · Cited by 200 · see citing works

Gene Therapy Restores Vision-Dependent Behavior as Well as Retinal Structure and Function in a Mouse Model of RPE65 Leber Congenital Amaurosis

Pang, J., Chang, B., Kumar, A., Nusinowitz, S., Noorwez, S. M., Li, J., Rani, A., Foster, T. C., Chiodo, V. A., Doyle, T. J., et al. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.09.001 · Cited by 199 · see citing works

Real-time, noninvasive in vivo assessment of adeno-associated virus-mediated retinal transduction.

Bennett, J., Duan, D., Engelhardt, J. F., & Maguire, A. M. (1997) · PubMed · Cited by 191 · see citing works

Prolonged Recovery of Retinal Structure/Function after Gene Therapy in an Rs1h-Deficient Mouse Model of X-Linked Juvenile Retinoschisis

Min, S.-H., Molday, L. L., Seeliger, M. W., Dinculescu, A., Timmers, A. M., Janßen, A., Tonagel, F., Tanimoto, N., Weber, B. H. F., Molday, R. S., et al. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.06.002 · Cited by 174 · see citing works

Persistent transgene product in retina, optic nerve and brain after intraocular injection of rAAV

Dudus, L., Anand, V., Acland, G. M., Chen, S.-J., Wilson, J. M., Fisher, K. J., Maguire, A. M., & Bennett, J. (1999) · Vision Research · doi:10.1016/s0042-6989(98)00308-3 · Cited by 160 · see citing works

Gene delivery to the eye using adeno-associated viral vectors

Martin, K. R. G., Klein, R. L., & Quigley, H. A. (2002) · Methods · doi:10.1016/s1046-2023(02)00232-3 · Cited by 149 · see citing works

Pharmacological and rAAV Gene Therapy Rescue of Visual Functions in a Blind Mouse Model of Leber Congenital Amaurosis

Batten, M. L., Imanishi, Y., Tu, D. C., Doan, T., Zhu, L., Pang, J., Glushakova, L., Moise, A. R., Baehr, W., Van Gelder, R. N., et al. (2005) · PLoS Medicine · doi:10.1371/journal.pmed.0020333 · Cited by 134 · see citing works

Gene Replacement Therapy Rescues Photoreceptor Degeneration in a Murine Model of Leber Congenital Amaurosis Lacking RPGRIP

Pawlyk, B. S., Smith, A. J., Buch, P. K., Adamian, M., Hong, D.-H., Sandberg, M. A., Ali, R. R., & Li, T. (2005) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.05-0371 · Cited by 126 · see citing works

Reporter Expression Persists 1 Year After Adeno-Associated Virus-Mediated Gene Transfer to the Optic Nerve

Guy, J. (1999) · Archives of Ophthalmology · doi:10.1001/archopht.117.7.929 · Cited by 114 · see citing works

Long-term doxycycline-regulated transgene expression in the retina of nonhuman primates following subretinal injection of recombinant AAV vectors

Stieger, K., Le Meur, G., Lasne, F., Weber, M., Deschamps, J.-Y., Nivard, D., Mendes-Madeira, A., Provost, N., Martin, L., Moullier, P., et al. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2005.12.001 · Cited by 110 · see citing works

A reliable method for organ culture of neonatal mouse retina with long-term survival

Ogilvie, J. M., Speck, J. D., Lett, J. M., & Fleming, T. T. (1999) · Journal of Neuroscience Methods · doi:10.1016/s0165-0270(98)00157-5 · Cited by 107 · see citing works

Adeno-Associated Virus-Vectored Gene Therapy for Retinal Disease

Dinculescu, A., Glushakova, L. G., Min, S.-H., & Hauswirth, W. W. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.649 · Cited by 101 · see citing works

Effect of deletions in adenovirus early region 1 genes upon replication of adeno-associated virus

LAUGHLIN, C. A., Jones, N. C., & Carter, B. J. (1982) · Journal of Virology · doi:10.1128/jvi.41.3.868-876.1982 · Cited by 77 · see citing works

Light-Driven Cone Arrestin Translocation in Cones of Postnatal Guanylate Cyclase-1 Knockout Mouse Retina Treated with AAV-GC1

Haire, S. E., Pang, J., Boye, S. L., Sokal, I., Craft, C. M., Palczewski, K., Hauswirth, W. W., & Semple‐Rowland, S. L. (2006) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.06-0086 · Cited by 66 · see citing works

Adeno-Associated Virus: A Ubiquitous Commensal of Mammals

Flotte, T. R., & Berns, K. I. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.401 · Cited by 61 · see citing works

Kinetics of transgene expression in mouse retina following sub-retinal injection of recombinant adeno-associated virus

Sarra, G., Stephens, C., Schlichtenbrede, F. C., Bainbridge, J. W., Thrasher, A. J., Luthert, P. J., & Ali, R. R. (2002) · Vision Research · doi:10.1016/s0042-6989(01)00230-9 · Cited by 58 · see citing works

Stable RAAV-Mediated Transduction of Rod and Cone Photoreceptors in the Canine Retina

Bainbridge, J. W. B., Mistry, A., Schlichtenbrede, F. C., Smith, A., Broderick, C., De Alwis, M., Georgiadis, A., Taylor, P. M., Squires, M., Sethi, C., et al. (2003) · Gene Therapy · doi:10.1038/sj.gt.3301990 · Cited by 58 · see citing works

Retrovirus-mediated gene transfer to retinal explants

Hatakeyama, J., & Kageyama, R. (2002) · Methods · doi:10.1016/s1046-2023(02)00257-8 · Cited by 55 · see citing works

Pseudotyped AAV vectors for constitutive and regulated gene expression in the eye

Auricchio, A. (2003) · Vision Research · doi:10.1016/s0042-6989(02)00676-4 · Cited by 50 · see citing works

AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice

Hennig, A. K., Ogilvie, J. M., Ohlemiller, K. K., Timmers, A. M., Hauswirth, W. W., & Sands, M. S. (2004) · Molecular Therapy · doi:10.1016/j.ymthe.2004.03.018 · Cited by 49 · see citing works

Delivery of Adeno-Associated Virus Vectors to the Fetal Retina: Impact of Viral Capsid Proteins on Retinal Neuronal Progenitor Transduction

Surace, E. M., Auricchio, A., Reich, S. J., Rex, T., Glover, E., Pineles, S., Tang, W., O'Connor, E., Lyubarsky, A., Savchenko, A., et al. (2003) · Journal of Virology · doi:10.1128/jvi.77.14.7957-7963.2003 · Cited by 46 · see citing works

Efficiency of lentiviral transduction during development in normal and rd mice.

Pang, J., Cheng, M., Haire, S. E., Barker, E., Planelles, V., & Blanks, J. C. (2006) · PubMed · Cited by 43 · see citing works

Adenoviral-mediated gene transfer to retinal explants during development and degeneration

Pang, J., Cheng, M., Stevenson, D., Trousdale, M. D., Dorey, C. K., & Blanks, J. C. (2004) · Experimental Eye Research · doi:10.1016/j.exer.2004.03.010 · Cited by 25 · see citing works

Adeno-Associated Virus-Mediated Expression of Vascular Endothelial Growth Factor Peptides Inhibits Retinal Neovascularization in a Mouse Model of Oxygen-Induced Retinopathy

Deng, W., Yan, Z., Dinculescu, A., Pang, J., Teusner, J. T., Cortez, N. G., Berns, K. I., & Hauswirth, W. W. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.1247 · Cited by 15 · see citing works

Müller Cell Transduction by AAV2 in Normal and Degenerative Retinas

Liang, F.-Q., Surace, E. M., Dejneka, N. S., Albert, M. M., & Bennett, J. (2003) · Advances in experimental medicine and biology · doi:10.1007/978-1-4615-0067-4_56 · Cited by 5 · see citing works

The mTORC1 effectors S6K1 and 4E-BP play different roles in CNS axon regeneration

Liu, Y., Miao, L., Liang, F., Huang, H., Teng, X., Li, S., Nuriddinov, J., Selzer, M. E., & Hu, Y. (2014) · Nature Communications · doi:10.1038/ncomms6416 · Cited by 158 · see citing works

AAV-Mediated Gene Therapy for Retinal Degeneration in the rd10 Mouse Containing a Recessive PDEβ Mutation

Pang, J., Boye, S. L., Kumar, A., Dinculescu, A., Deng, W., Li, J., Li, Q., Rani, A., Foster, T. C., Chang, B., et al. (2008) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.07-1622 · Cited by 137 · see citing works

In vitro and ex vivo models to study drug delivery barriers in the posterior segment of the eye

Peynshaert, K., Devoldere, J., Smedt, S. C. D., & Remaut, K. (2017) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2017.09.007 · Cited by 133 · see citing works

AAV integration in human hepatocytes

Dalwadi, D. A., Calabria, A., Tiyaboonchai, A., Posey, J., Naugler, W. E., Montini, E., & Grompe, M. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.08.031 · Cited by 126 · see citing works

mTORC1 is necessary but mTORC2 and GSK3β are inhibitory for AKT3-induced axon regeneration in the central nervous system

Miao, L., Liu, Y., Huang, H., Liang, F., Ling, C., & Hu, Y. (2016) · eLife · doi:10.7554/elife.14908 · Cited by 122 · see citing works

Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus

Lopes, V. S., Boye, S. E., Louie, C. M., Boye, S., Dyka, F., Chiodo, V, Fofo, H., Hauswirth, W. W., & Williams, D. S. (2013) · Gene Therapy · doi:10.1038/gt.2013.3 · Cited by 112 · see citing works

rAAV2/5 gene-targeting to rods:dose-dependent efficiency and complications associated with different promoters

Beltran, W. A., Boye, S. L., Boye, S. E., Chiodo, V. A., Lewin, A. S., Hauswirth, W. W., & Aguirre, G. D. (2010) · Gene Therapy · doi:10.1038/gt.2010.56 · Cited by 96 · see citing works

Subretinal injection techniques for retinal disease: A review

Irigoyen, C., Amenabar Alonso, A., Sanchez-Molina, J., Rodríguez-Hidalgo, M., Lara-López, A., & Ruiz-Ederra, J. (2022) · Journal of Clinical Medicine · doi:10.3390/jcm11164717 · Cited by 106 · see citing works

Ultrasound-targeted microbubble destruction enhances AAV-mediated gene transfection in human RPE cells in vitro and rat retina in vivo

Li, H. L., Zheng, X. Z., Wang, H. P., Li, F., Wu, Y., & Du, L. (2009) · Gene Therapy · doi:10.1038/gt.2009.84 · Cited by 86 · see citing works

Mouse γ-Synuclein Promoter-Mediated Gene Expression and Editing in Mammalian Retinal Ganglion Cells

Qizhao, W., Zhuang, P., Huang, H., Li, L., Liu, L., Webber, H. C., Dalal, R., Siew, L., Fligor, C. M., Chang, K., et al. (2020) · Journal of Neuroscience · doi:10.1523/jneurosci.0102-20.2020 · Cited by 79 · see citing works

Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters

Nieuwenhuis, B., Laperrousaz, E., Tribble, J. R., Verhaagen, J., Fawcett, J. W., Martin, K. R. G., Williams, P. A., & Osborne, A. (2023) · Gene Therapy · doi:10.1038/s41434-022-00380-z · Cited by 77 · see citing works

Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency

Pang, J., Boye, S. L., Lei, B., Boye, S. L., Everhart, D., Ryals, R. C., Umino, Y., Rohrer, B., Alexander, J. J., Li, J., et al. (2010) · Gene Therapy · doi:10.1038/gt.2010.29 · Cited by 70 · see citing works

Optic nerve regeneration screen identifies multiple genes restricting adult neural repair

Lindborg, J. A., Tran, N. M., Chenette, D. M., DeLuca, K., Foli, Y., Kannan, R., Sekine, Y., Wang, X., Wollan, M., Kim, I.-J., et al. (2021) · Cell Reports · doi:10.1016/j.celrep.2021.108777 · Cited by 70 · see citing works

LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile

Koilkonda, R., Yu, H., Talla, V., Porciatti, V., Feuer, W. J., Hauswirth, W. W., Chiodo, V., Erger, K. E., Boye, S. L., Lewin, A. S., et al. (2014) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.14-15388 · Cited by 65 · see citing works

In vivo genome editing as a potential treatment strategy for inherited retinal dystrophies

Yanik, M., Müller, B., Song, F., Gáll, J., Wagner, F., Wende, W., Lorenz, B., & Stieger, K. (2016) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2016.09.001 · Cited by 67 · see citing works

Evaluation of the adeno-associated virus mediated long-term expression of channelrhodopsin-2 in the mouse retina.

Ivanova, E., & Pan, Z. (2009) · PubMed Central · Cited by 66 · see citing works

Gene Therapy Rescues Cone Structure and Function in the 3-Month-Old rd12 Mouse: A Model for Midcourse RPE65 Leber Congenital Amaurosis

Li, X., Li, W., Dai, X., Kong, F., Zheng, Q., Zhou, X., Lü, F., Chang, B., Rohrer, B., Hauswirth, W. W., et al. (2010) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.10-6138 · Cited by 65 · see citing works

Ab-Externo AAV-Mediated Gene Delivery to the Suprachoroidal Space Using a 250 Micron Flexible Microcatheter

Peden, M. C., Min, J., Meyers, C., Lukowski, Z., Li, Q., Boye, S. L., Levine, M., Hauswirth, W. W., Ratnakaram, R., Dawson, W., et al. (2011) · PLoS ONE · doi:10.1371/journal.pone.0017140 · Cited by 63 · see citing works

Retinal tropism and transduction of adeno-associated virus varies by serotype and route of delivery (intravitreal, subretinal, or suprachoroidal) in rats

Han, I. C., Cheng, J. L., Burnight, E. R., Ralston, C. L., Fick, J. L., Thomsen, G. J., Tovar, E. F., Russell, S. R., Sohn, E. H., Mullins, R. F., et al. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.043 · Cited by 60 · see citing works

Review: the history and role of naturally occurring mouse models with Pde6b mutations.

Han, J., Dinculescu, A., Dai, X., Du, W., Smith, W. C., & Pang, J. (2013) · Europe PMC (PubMed Central) · Cited by 61 · see citing works

Neuroprotection by eIF2α-CHOP inhibition and XBP-1 activation in EAE/optic neuritiss

Huang, H., Miao, L., Liang, F., Liu, X., Xu, L., Teng, X., Qizhao, W., Ridder, W. H., Shindler, K. S., Sun, Y., et al. (2017) · Cell Death and Disease · doi:10.1038/cddis.2017.329 · Cited by 62 · see citing works

AAV-mediated base-editing therapy ameliorates the disease phenotypes in a mouse model of retinitis pigmentosa

Wu, Y., Wan, X., Zhao, D., Chen, X., Wang, Y., Tang, X., Li, J., Li, S., Sun, X., Bi, C., et al. (2023) · Nature Communications · doi:10.1038/s41467-023-40655-6 · Cited by 58 · see citing works

Trans-Corneal Subretinal Injection in Mice and Its Effect on the Function and Morphology of the Retina

Qi, Y., Dai, X., Zhang, H., He, Y., Zhang, Y., Han, J., Zhu, P., Zhang, Y., Zheng, Q., Li, X., et al. (2015) · PLoS ONE · doi:10.1371/journal.pone.0136523 · Cited by 55 · see citing works

Tyrosine-Mutant AAV8 Delivery of Human MERTK Provides Long-Term Retinal Preservation in RCS Rats

Deng, W., Dinculescu, A., Li, Q., Boye, S. L., Li, J., Gorbatyuk, M. S., Pang, J., Chiodo, V. A., Matthes, M. T., Yasumura, D., et al. (2012) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.11-8831 · Cited by 55 · see citing works

Self-complementary AAV5 vector facilitates quicker transgene expression in photoreceptor and retinal pigment epithelial cells of normal mouse

Kong, F., Li, W., Li, X., Zheng, Q., Dai, X., Zhou, X., Boye, S. L., Hauswirth, W. W., Qu, J., & Pang, J. (2010) · Experimental Eye Research · doi:10.1016/j.exer.2010.01.011 · Cited by 52 · see citing works

Gene therapy for diabetic retinopathy: Are we ready to make the leap from bench to bedside?

Wang, J.-H., Ling, D. L., Tu, L., van Wijngaarden, P., Dusting, G. J., & Liu, G. (2017) · Pharmacology & Therapeutics · doi:10.1016/j.pharmthera.2017.01.003 · Cited by 51 · see citing works

Update on viral gene therapy clinical trials for retinal diseases

Cheng, S.-Y., & Punzo, C. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.159 · Cited by 49 · see citing works

XIAP Therapy Increases Survival of Transplanted Rod Precursors in a Degenerating Host Retina

Yao, J., Feathers, K. L., Khanna, H., Thompson, D. A., Tsilfidis, C., Hauswirth, W. W., Heckenlively, J. R., Swaroop, A., & Zacks, D. N. (2010) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.10-5998 · Cited by 50 · see citing works

The use of canine models of inherited retinal degeneration to test novel therapeutic approaches

Beltran, W. A. (2009) · Veterinary Ophthalmology · doi:10.1111/j.1463-5224.2009.00694.x · Cited by 47 · see citing works

Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye.

Li, W., Kong, F., Li, X., Dai, X., Liu, X., Zheng, Q., Wu, R., Zhou, X., Lü, F., Chang, B., et al. (2009) · PubMed Central · Cited by 45 · see citing works

Organotypic Cultures of Adult Mouse Retina: Morphologic Changes and Gene Expression

Müller, B., Wagner, F., Lorenz, B., & Stieger, K. (2017) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.16-20718 · Cited by 44 · see citing works

Progress in Gene Therapy to Prevent Retinal Ganglion Cell Loss in Glaucoma and Leber’s Hereditary Optic Neuropathy

Ratican, S., Osborne, A., & Martin, K. R. G. (2018) · Neural Plasticity · doi:10.1155/2018/7108948 · Cited by 44 · see citing works

Gene therapy and transplantation in the retinofugal pathway

Harvey, A. R., Hellström, M., & Rodger, J. (2009) · Progress in brain research · doi:10.1016/s0079-6123(09)17510-6 · Cited by 42 · see citing works

Delivery of CR2-fH Using AAV Vector Therapy as Treatment Strategy in the Mouse Model of Choroidal Neovascularization

Schnabolk, G., Parsons, N., Obert, E., Annamalai, B., Nasarre, C., Tomlinson, S., Lewin, A. S., & Rohrer, B. (2017) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2017.11.003 · Cited by 40 · see citing works

Comparative AAV-eGFP Transgene Expression Using Vector Serotypes 1–9, 7m8, and 8b in Human Pluripotent Stem Cells, RPEs, and Human and Rat Cortical Neurons

Duong, T. T., Lim, J., Vasireddy, V., Papp, T., Nguyen, H., Leo, L., Pan, J., Zhou, S., Chen, H. I., Bennett, J., et al. (2019) · Stem Cells International · doi:10.1155/2019/7281912 · Cited by 33 · see citing works

In vivo imaging of adeno-associated viral vector labelled retinal ganglion cells

Smith, C. A., & Chauhan, B. C. (2018) · Scientific Reports · doi:10.1038/s41598-018-19969-9 · Cited by 34 · see citing works

A Novel Method Combining Vitreous Aspiration and Intravitreal AAV2/8 Injection Results in Retina-Wide Transduction in Adult Mice

Costa, R. D., Röger, C., Segelken, J., Barben, M., Grimm, C., & Neidhardt, J. (2016) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.16-19701 · Cited by 31 · see citing works

Gene Therapy for Inherited Retinal Degeneration

Arbabi, A., Liu, A., & Ameri, H. (2019) · Journal of Ocular Pharmacology and Therapeutics · doi:10.1089/jop.2018.0087 · Cited by 30 · see citing works

Development of a Pde6b Gene Knockout Rat Model for Studies of Degenerative Retinal Diseases

Yeo, J. H., Jung, B. K., Lee, H., Baek, I., Sung, Y. H., Shin, H.-S., Kim, H. K., Seo, K. Y., & Lee, J. Y. (2019) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.18-25556 · Cited by 28 · see citing works

Dre - Cre Sequential Recombination Provides New Tools for Retinal Ganglion Cell Labeling and Manipulation in Mice

Sajgo, S., Tegla, M. G. G., Shi, M., Liu, P., Dong, L., Parmhans, N., Popescu, O., & Badea, T. C. (2014) · PLoS ONE · doi:10.1371/journal.pone.0091435 · Cited by 27 · see citing works

Rod Outer Segment Development Influences AAV-Mediated Photoreceptor Transduction After Subretinal Injection

Petit, L., Ma, S., Cheng, S.-Y., Gao, G., & Punzo, C. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.020 · Cited by 24 · see citing works

Continuous Affinity Purification of Adeno-Associated Virus Using Periodic Counter-Current Chromatography

Mendes, J. P., Bergman, M., Solbrand, A., Peixoto, C., Carrondo, M. J. T., & Silva, R. J. S. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14071346 · Cited by 23 · see citing works

Cone Phosphodiesterase-6 ' Restores Rod Function and Confers Distinct Physiological Properties in the Rod Phosphodiesterase-6 -Deficient rd10 Mouse

Deng, W., Sakurai, K., Kolandaivelu, S., Kolesnikov, A. V., Dinculescu, A., Li, J., Zhu, P., Liu, X., Pang, J., Chiodo, V. A., et al. (2013) · Journal of Neuroscience · doi:10.1523/jneurosci.1536-13.2013 · Cited by 23 · see citing works

AAV2 and AAV9 tropism and transgene expression in the mouse eye and major tissues after intravitreal and subretinal delivery

Koponen, S., Kokki, E., Tamminen, T., & Ylä-Herttuala, S. (2023) · Frontiers in Drug Delivery · doi:10.3389/fddev.2023.1148795 · Cited by 21 · see citing works

Seeing the Future: A Review of Ocular Therapy

Whalen, M., Akula, M., McNamee, S. M., DeAngelis, M. M., & Haider, N. B. (2024) · Bioengineering · doi:10.3390/bioengineering11020179 · Cited by 21 · see citing works

The oncomodulin receptor ArmC10 enables axon regeneration in mice after nerve injury and neurite outgrowth in human iPSC–derived sensory neurons

Xie, L., Yin, Y., Jayakar, S. S., Kawaguchi, R., Wang, Q., Peterson, S. L., Shi, C., Turnes, B. L., Zhang, Z., Osés-Prieto, J. A., et al. (2023) · Science Translational Medicine · doi:10.1126/scitranslmed.adg6241 · Cited by 20 · see citing works

A novel approach to attenuate proliferative vitreoretinopathy using ultrasound‐targeted microbubble destruction and recombinant adeno‐associated virus‐mediated RNA interference targeting transforming growth factor‐β2 and platelet‐derived growth factor‐B

Zheng, X.-Z., Du, L., Wang, H., & Gu, Q. (2012) · The Journal of Gene Medicine · doi:10.1002/jgm.2629 · Cited by 20 · see citing works

Long-term retinal cone rescue using a capsid mutant AAV8 vector in a mouse model of CNGA3-achromatopsia

Dai, X., He, Y., Zhang, H., Zhang, Y., Liu, Y., Wang, M., Chen, H., & Pang, J. (2017) · PLoS ONE · doi:10.1371/journal.pone.0188032 · Cited by 20 · see citing works

Erythropoietin Gene Therapy Delays Retinal Degeneration Resulting from Oxidative Stress in the Retinal Pigment Epithelium

Biswal, M. R., Wang, Z., Paulson, R. J., Uddin, R. R., Tong, Y., Zhu, P., Li, H., & Lewin, A. S. (2021) · Antioxidants · doi:10.3390/antiox10060842 · Cited by 19 · see citing works

A Novel Experimental Mouse Model of Retinal Detachment: Complete Functional and Histologic Recovery of the Retina

Zeng, R., Zhang, Y., Shi, F., & Kong, F. (2012) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.11-8241 · Cited by 18 · see citing works

AAV-Mediated Gene Therapy in Mouse Models of Recessive Retinal Degeneration

Pang, J., Lei, L., Dai, X., Shi, W., Liu, X., Dinculescu, A., & McDowell, J. H. (2012) · Current Molecular Medicine · doi:10.2174/156652412799218877 · Cited by 17 · see citing works

In vivo cellular imaging of various stress/response pathways using AAV following axonal injury in mice

Fujita, K., Nishiguchi, K. M., Yokoyama, Y., Tomiyama, Y., Tsuda, S., Yasuda, M., Maekawa, S., & Nakazawa, T. (2015) · Scientific Reports · doi:10.1038/srep18141 · Cited by 18 · see citing works

Subretinal Rather Than Intravitreal Adeno-Associated Virus-Mediated Delivery of a Complement Alternative Pathway Inhibitor Is Effective in a Mouse Model of RPE Damage

Annamalai, B., Parsons, N., Nicholson, C., Obert, E., Jones, B., & Rohrer, B. (2021) · Investigative Opthalmology & Visual Science · doi:10.1167/iovs.62.4.11 · Cited by 16 · see citing works

Enhanced transduction and improved photoreceptor survival of retinal degeneration by the combinatorial use of rAAV2 with a lower dose of adenovirus

Wu, J., Zhang, S., Wu, X., Dong, X., Xu, P., Liu, X., Li, C., & Huang, Q. (2008) · Vision Research · doi:10.1016/j.visres.2008.04.019 · Cited by 16 · see citing works

Organotypic Culture of Adult Mouse Retina

Müller, B. (2019) · Methods in molecular biology · doi:10.1007/978-1-4939-9086-3_13 · Cited by 14 · see citing works

Adeno Associated Virus (AAV) as a Tool for Clinical and Experimental Delivery of Target Genes into the Mammalian Retina

Hori, T., Fukutome, M., & Koike, C. (2019) · Biological and Pharmaceutical Bulletin · doi:10.1248/bpb.b18-00913 · Cited by 15 · see citing works

Optimized Subretinal Injection Technique for Gene Therapy Approaches

Mühlfriedel, R., Michalakis, S., Garrido, M. G., Sothilingam, V., Schön, C., Biel, M., & Seeliger, M. W. (2019) · Methods in Molecular Biology · doi:10.1007/978-1-4939-8669-9_26 · Cited by 14 · see citing works

An optimized protocol for generating labeled and transplantable photoreceptor precursors from human embryonic stem cells

Markus, A., Shamul, A., Chemla, Y., Farah, N., Shaham, L., Goldstein, R. S., & Mandel, Y. (2018) · Experimental Eye Research · doi:10.1016/j.exer.2018.11.013 · Cited by 14 · see citing works

In Vivo Evaluation of PAX6 Overexpression and NMDA Cytotoxicity to Stimulate Proliferation in the Mouse Retina

Pirmardan, E. R., Soheili, Z., Samiei, S., Ahmadieh, H., Mowla, S. J., Naseri, M., & Daftarian, N. (2018) · Scientific Reports · doi:10.1038/s41598-018-35884-5 · Cited by 14 · see citing works

Can an in vivo imaging system be used to determine localization and biodistribution of AAV5-mediated gene expression following subretinal and intravitreal delivery in mice?

Ezra-Elia, R., Obolensky, A., Ejzenberg, A., Ross, M., Mintz, D., Banin, E., & Ofri, R. (2018) · Experimental Eye Research · doi:10.1016/j.exer.2018.08.021 · Cited by 13 · see citing works

NHE8 is essential for RPE cell polarity and photoreceptor survival

Xia, C.-H., Liu, H., Cheung, D., Tang, F., Chang, B., Li, M., & Gong, X. (2015) · Scientific Reports · doi:10.1038/srep09358 · Cited by 12 · see citing works

Phosphorylated S6K1 and 4E-BP1 play different roles in constitutively active Rheb-mediated retinal ganglion cell survival and axon regeneration after optic nerve injury

Jiang, J., Zhang, L., Zou, J., Liu, J., Yang, J., Jiang, Q., Duan, P., & Jiang, B. (2023) · Neural Regeneration Research · doi:10.4103/1673-5374.371372 · Cited by 11 · see citing works

Gene delivery to cone photoreceptors by subretinal injection of rAAV2/6 in the mouse retina

Hori, T., Fukutome, M., Maejima, C., Matsushima, H., Kobayashi, K., Kitazawa, S., Kitahara, R., Kitano, K., Kobayashi, K., Moritoh, S., et al. (2019) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2019.05.117 · Cited by 11 · see citing works

In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans

Schwartz, M. K., Likhite, S. B., Vetter, T. A., Baird, M., McGovern, V. L., Sierra-Delgado, J. A., Mendel, T., Burghes, A. H. M., & Meyer, K. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.05.016 · Cited by 10 · see citing works

Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model RhoP23H+/−

McNamee, S. M., Chan, N. P., Akula, M., Avola, M. O., Whalen, M., Nystuen, K., Singh, P., Upadhyay, A. K., DeAngelis, M. M., & Haider, N. B. (2024) · Gene Therapy · doi:10.1038/s41434-024-00440-6 · Cited by 10 · see citing works

Identification of Key Residues Determining Isomerohydrolase Activity of Human RPE65

Takahashi, Y., Moiseyev, G. P., & Ma, J.-X. (2014) · Journal of Biological Chemistry · doi:10.1074/jbc.m114.558619 · Cited by 10 · see citing works

Comparative Analysis of Gene Transfer to Human and Rat Retinal Pigment Epithelium Cell Line by a Combinatorial Use of Recombinant Adeno- Associated Virus and Ultrasound or/and Microbubbles

Zheng, X.-Z., Li, H., Du, L., Wang, H., & Gu, Q. (2009) · Bosnian Journal of Basic Medical Sciences · doi:10.17305/bjbms.2009.2802 · Cited by 10 · see citing works

β-secretase 1 overexpression by AAV-mediated gene delivery prevents retina degeneration in a mouse model of age-related macular degeneration

Qi, X., Francelin, C., Mitter, S. K., Boye, S. L., Gu, H., Quigley, J., Grant, M. B., & Boulton, M. E. (2023) · Molecular Therapy · doi:10.1016/j.ymthe.2023.03.029 · Cited by 9 · see citing works

In vivo and in vitro knockout system labelled using fluorescent protein via microhomology-mediated end joining

Katayama, S., Sato, K., & Nakazawa, T. (2019) · Life Science Alliance · doi:10.26508/lsa.201900528 · Cited by 9 · see citing works

Cone Phosphodiesterase-6γ’ Subunit Augments Cone PDE6 Holoenzyme Assembly and Stability in a Mouse Model Lacking Both Rod and Cone PDE6 Catalytic Subunits

Deng, W., Kolandaivelu, S., Dinculescu, A., Li, J., Zhu, P., Chiodo, V. A., Ramamurthy, V., & Hauswirth, W. W. (2018) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2018.00233 · Cited by 8 · see citing works

The Degeneration and Apoptosis Patterns of Cone Photoreceptors in rd11 Mice

Zhang, H., Li, X., Dai, X., Han, J., Zhang, Y., Qi, Y., He, Y., Liu, Y., Chang, B., & Pang, J. (2017) · Journal of Ophthalmology · doi:10.1155/2017/9721362 · Cited by 7 · see citing works

Peripheral cellular immune responses induced by subretinal adeno-associated virus gene transfer can Be restrained by the subretinal-associated immune inhibition mechanism

Vendomèle, J., Chauveau, G. A., Dalkara, D., Galy, A., & Fisson, S. (2024) · Human Gene Therapy · doi:10.1089/hum.2023.191 · Cited by 6 · see citing works

Regulatable Complement Inhibition of the Alternative Pathway Mitigates Wet Age-Related Macular Degeneration Pathology in a Mouse Model

Parsons, N., Annamalai, B., & Rohrer, B. (2023) · Translational Vision Science & Technology · doi:10.1167/tvst.12.7.17 · Cited by 6 · see citing works

Genersatztherapie bei genetisch bedingter Zapfenblindheit

Mühlfriedel, R. L., Tanimoto, N., & Seeliger, M. W. (2014) · Klinische Monatsblätter für Augenheilkunde · doi:10.1055/s-0034-1368180 · Cited by 7 · see citing works

Intravitreal Injection of AAV for the Transduction of Mouse Retinal Ganglion Cells

Nieuwenhuis, B., & Osborne, A. (2023) · Methods in molecular biology · doi:10.1007/978-1-0716-3409-7_17 · Cited by 4 · see citing works

Marcks overexpression in retinal ganglion cells promotes optic nerve regeneration

Peng, X.-Q., Li, Y.-Z., Gu, C., He, X., Li, C.-P., Yong-quan, S., Du, H., Teng, Z., & Liu, C. (2024) · Cell Death and Disease · doi:10.1038/s41419-024-07281-6 · Cited by 5 · see citing works

The change of microglia numbers within the mice retina, optic nerve and chiasm following intravitreal AAV2-GFP injection

Ji, Y., Yu, B., Zhang, Y., & Wu, W. (2022) · European Journal of Ophthalmology · doi:10.1177/11206721211049001 · Cited by 3 · see citing works

Accelerated maturation of ARPE ‐19 cells for the translational assessment of gene therapy

Bernd, J., Plastino, F., Karayannis, J. J., Kvanta, A., Locri, F., & André, H. (2024) · The FASEB Journal · doi:10.1096/fj.202301707rr · Cited by 3 · see citing works

Semiquantitative Methods for GFP Immunohistochemistry and In Situ Hybridization to Evaluate AAV Transduction of Mouse Retinal Cells Following Subretinal Injection

Bondarenko, G. I., Sorden, S. D., Christian, B. J., Webster, S., & Sharma, A. K. (2020) · Toxicologic Pathology · doi:10.1177/0192623320964804 · Cited by 3 · see citing works

Evaluating therapeutic potential of NR2E3 doses in the rd7 mouse model of retinal degeneration

McNamee, S. M., Akula, M., Love, Z., Nasraty, N., Nystuen, K., Singh, P., Upadhyay, A. K., DeAngelis, M. M., & Haider, N. B. (2024) · Scientific Reports · doi:10.1038/s41598-024-67095-6 · Cited by 3 · see citing works

A synthetic circular RNA targeting miR-340-5p promotes optic nerve regeneration and retinal ganglion cell survival following axotomy

Hintermayer, M. A., Hua, E. M.-L., Noor, M., Rambaldi, I., & Fournier, A. E. (2025) · Experimental Neurology · doi:10.1016/j.expneurol.2025.115527 · Cited by 2 · see citing works

Genersatztherapie bei hereditären Netzhauterkrankungen

Mühlfriedel, R. L., Sothilingam, V., Tanimoto, N., & Seeliger, M. W. (2017) · Klinische Monatsblätter für Augenheilkunde · doi:10.1055/s-0043-101817 · Cited by 2 · see citing works

The potential use of anti-codon engineered tRNAs (ACE-tRNAs) to treat nonsense variants causing inherited retinal diseases

Sarma, A. S., & Sharon, D. (2025) · Molecular Aspects of Medicine · doi:10.1016/j.mam.2025.101443 · Cited by 1 · see citing works

Intravitreal injection of new adeno-associated viral vector: Enhancing retinoschisin 1 gene transduction in a mouse model of X-linked retinoschisis

Sun, Y., Xiao, D., Li, Z., Xu, D., Zhang, D., An, Y., Xue, J., Ren, Y., Liu, S., Wang, D., et al. (2024) · Biochemistry and Biophysics Reports · doi:10.1016/j.bbrep.2024.101646 · Cited by 1 · see citing works

In vivo imaging of the light response in mouse retinal ganglion cells based on a neuronal activity-dependent promoter

Fujita, K., Nishiguchi, K. M., Sato, K., Nakagawa-Saito, Y., & Nakazawa, T. (2019) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2019.10.155 · Cited by 1 · see citing works

Transcriptional regulation of the rod-specific transcription factor, Nrl

Kautzmann, M. A. (2012) · theses.fr (ABES) · doi:10.70675/33b247a9z44a9z477eza84dz15857187975a

Genetic Modification of Adeno-Associated Virus Capsid for Enhanced Motor Neuron Delivery and Retrograde Transport

Davis, A. (2012) · OhioLink ETD Center (Ohio Library and Information Network)

Restoring Visual Function After Photoreceptor Degeneration: Ectopic Expression of Photosensitive Proteins in Retinal Neurons

Lin, B., & Masland, R. H. (2011) · Neuromethods · doi:10.1007/978-1-61779-031-7_8

The Role of Stat3 in Mouse Models of Inherited Photoreceptor Degeneration

Jiang, K. (2013) · TSpace (University of Toronto)

Molecular insights into PCARE-associated retinal disease

Serrano, J. C. C. (2018) · Radboud Repository (Radboud University)

DETERMINANTS OF AAV TRANSPORT ACROSS THE BLOOD-BRAIN BARRIER

Albright, B. H. (2019) · Carolina Digital Repository (University of North Carolina at Chapel Hill) · doi:10.17615/vnpr-wx33

Cellular Immune Responses Induced by Subretinal AAV Gene Transfer can be Restrained by the Subretinal Associated Immune Inhibition MechanismPreprint

Vendomèle, J., Chauveau, G. A., Dalkara, D., Galy, A. H. M., & Fisson, S. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2022.12.30.522324

Gene Therapy and Gene Editing

Assaf, B. T., Harper, C. G., & Phillips, J. A. (2023) · Elsevier eBooks · doi:10.1016/b978-0-12-821047-5.00034-8

Gene Expression and Editing in Mammalian Retinal Ganglion Cells

Tran, N. M., Xu, X., Qi, L. S., & Hu, Y. (2024) · Essentials in ophthalmology/Essentials ophthalmology · doi:10.1007/978-981-99-4436-1_19

Optic nerve regeneration requires the intracellular domain of LIFRa/CD118Preprint

Jiang, Q., Wang, C., Ren, Y., Duan, P., tian, K., Duan, X., Cai, B., Xu, C., Li, J., Benowitz, L. I., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.10.14.682231

Lipid nanoparticles enable mRNA delivery to diverse cell types of the inner Retina

Biswas, S., Carpentiero, E., Xhaferri, N., Streckenbach, P., Renigunta, V., & Lindner, M. (2026) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2026.102996

Optimized AAV5-RPGR ORF15 Gene Therapy Rescues Photoreceptor Structure and Function in X-Linked Retinitis Pigmentosa Mouse Model

Chen, X., Xu, X., Cao, S., Chunyan, P., Wei, S., 侯豹可, & Zhou, H. (2026) · American Journal of Ophthalmology · doi:10.1016/j.ajo.2026.06.005

Tropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina

Hickey, D. G., Edwards, T. L., Barnard, A. R., Singh, M. S., de Silva, S. R., McClements, M. E., Flannery, J. G., Hankins, M. W., & MacLaren, R. E. (2017) · Gene Therapy · doi:10.1038/gt.2017.85 · Cited by 72 · see citing works

Tropisms of AAV for subretinal delivery to the neonatal mouse retina and its application for in vivo rescue of developmental photoreceptor disorders. PLoS One 8

Watanabe, S., Sanuki, R., Ueno, S., Koyasu, T., Hasegawa, T., & Furukawa, T. (2013)

Transduction of the inner mouse retina using AAVrh8 and AAVrh10 via intravitreal injection

Giove, T. J., Sena-Esteves, M., & Eldred, W. D. (2010) · Experimental Eye Research · doi:10.1016/j.exer.2010.08.011 · Cited by 27 · see citing works

Tropisms of AAV for Subretinal Delivery to the Neonatal Mouse Retina and Its Application for In Vivo Rescue of Developmental Photoreceptor Disorders

Watanabe, S., Sanuki, R., Ueno, S., Koyasu, T., Hasegawa, T., & Furukawa, T. (2013) · PLoS ONE · doi:10.1371/journal.pone.0054146 · Cited by 59 · see citing works

Tropism and Retinal Transduction Efficiency of Adeno-Associated Virus Serotypes in Mice

Wu, W., McRae, J., Brown, A., & Agbaga, M. (2025) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.66.12.18 · Cited by 2 · see citing works