Clinical Applications Involving CNS Gene Transfer

In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector

Naldini, L., Blömer, U., Gallay, P., Ory, D., Mulligan, R., Gage, F. H., Verma, I. M., & Trono, D. (1996) · Science · doi:10.1126/science.272.5259.263 · Cited by 4,903 · see citing works

<i>LMO2</i> -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

Hacein-Bey-Abina, S., Von Kalle, C., Schmidt, M., McCormack, M. P., Wulffraat, N., Leboulch, P., Lim, A., Osborne, C. S., Pawliuk, R., Morillon, E., et al. (2003) · Science · doi:10.1126/science.1088547 · Cited by 3,587 · see citing works

A Third-Generation Lentivirus Vector with a Conditional Packaging System

Dull, T., Zufferey, R., Kelly, M., Mandel, R. J., Nguyen, M., Trono, D., & Naldini, L. (1998) · Journal of Virology · doi:10.1128/jvi.72.11.8463-8471.1998 · Cited by 3,341 · 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

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

Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo

Zufferey, R., Nagy, D., Mandel, R. J., Naldini, L., & Trono, D. (1997) · Nature Biotechnology · doi:10.1038/nbt0997-871 · Cited by 1,977 · 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

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

Nathwani, A. C., Tuddenham, E. G. D., Rangarajan, S., Rosales, C., McIntosh, J., Linch, D. C., Chowdary, P., Riddell, A., Pie, A. J., Harrington, C., et al. (2011) · New England Journal of Medicine · doi:10.1056/nejmoa1108046 · Cited by 1,864 · see citing works

Self-Inactivating Lentivirus Vector for Safe and Efficient In Vivo Gene Delivery

Zufferey, R., Dull, T., Mandel, R. J., Bukovsky, A., Quiroz, D., Naldini, L., & Trono, D. (1998) · Journal of Virology · doi:10.1128/jvi.72.12.9873-9880.1998 · Cited by 1,815 · see citing works

T Lymphocyte-Directed Gene Therapy for ADA − SCID: Initial Trial Results After 4 Years

Blaese, R. M., Culver, K. W., Miller, A. D., Carter, C. S., Fleisher, T. A., Clerici, M., Shearer, G. M., Chang, L., Chiang, Y. L., Tolstoshev, P., et al. (1995) · Science · doi:10.1126/science.270.5235.475 · Cited by 1,546 · see citing works

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. C., Veres, G., Schmidt, M., Kutschera, I., Vidaud, M., Abel, U., Dal-Cortivo, L., Caccavelli, L., et al. (2009) · Science · doi:10.1126/science.1171242 · Cited by 1,508 · see citing works

Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector.

Naldini, L., Blömer, U., Gage, F. H., Trono, D., & Verma, I. M. (1996) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.93.21.11382 · Cited by 1,499 · see citing works

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

Foust, K. D., Nurre, E., Montgomery, C. L., Hernandez, A., Chan, C. M., & Kaspar, B. K. (2009) · Nature Biotechnology · doi:10.1038/nbt.1515 · Cited by 1,456 · cited 4× · see citing works

Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection.

Miller, A. D., Adam, M., & Miller, A. D. (1990) · Molecular and Cellular Biology · doi:10.1128/mcb.10.8.4239 · Cited by 1,445 · see citing works

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

Cavazzana-Calvo, M., Payen, E., Negre, O., Wang, G., Hehir, K., Fusil, F., Down, J., Denaro, M., Brady, T., Westerman, K., et al. (2010) · Nature · doi:10.1038/nature09328 · Cited by 1,276 · see citing works

Development of a self-inactivating lentivirus vector

Miyoshi, H., Blömer, U., Takahashi, M., Gage, F. H., & Verma, I. M. (1998) · Journal of Virology · doi:10.1128/jvi.72.10.8150-8157.1998 · Cited by 1,196 · see citing works

Gene therapy clinical trials worldwide to 2012 – an update

Ginn, S. L., Alexander, I. E., Edelstein, M. L., Abedi, M. R., & Wixon, J. (2013) · The Journal of Gene Medicine · doi:10.1002/jgm.2698 · Cited by 1,186 · see citing works

The autophagy-related protein beclin 1 shows reduced expression in early Alzheimer disease and regulates amyloid beta accumulation in mice.

Pickford, F., Masliah, E., Britschgi, M., Lucin, K. M., Narasimhan, R., Jaeger, P. A., Small, S., Spencer, B., Rockenstein, E., Levine, B., et al. (2008) · Journal of Clinical Investigation · doi:10.1172/jci33585 · Cited by 1,186 · see citing works

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

Biffi, A., Montini, E., Lorioli, L., Cesani, M., Fumagalli, F., Plati, T., Baldoli, C., Martino, S., Calabria, A., Canale, S., et al. (2013) · Science · doi:10.1126/science.1233158 · Cited by 1,180 · see citing works

Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson’s disease: an open label, phase I trial

Kaplitt, M. G., Feigin, A., Tang, C., Fitzsimons, H. L., Mattis, P., Lawlor, P. A., Bland, R. J., Young, D., Strybing, K., Eidelberg, D., et al. (2007) · The Lancet · doi:10.1016/s0140-6736(07)60982-9 · Cited by 1,067 · see citing works

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

Aiuti, A., Biasco, L., Scaramuzza, S., Ferrua, F., Cicalese, M. P., Baricordi, C., Dionisio, F., Calabria, A., Giannelli, S., Castiello, M. C., et al. (2013) · Science · doi:10.1126/science.1233151 · Cited by 1,025 · see citing works

Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances Expression of Transgenes Delivered by Retroviral Vectors

Zufferey, R., Donello, J. E., Trono, D., & Hope, T. J. (1999) · Journal of Virology · doi:10.1128/jvi.73.4.2886-2892.1999 · Cited by 1,012 · see citing works

Adeno-associated Virus Serotypes: Vector Toolkit for Human Gene Therapy

Wu, Z., Asokan, A., & Samulski, R. J. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.05.009 · Cited by 921 · see citing works

HIV-1 genome nuclear import is mediated by a central DNA flap.

Zennou, V., Petit, C., Guétard, D., Nerhbass, U., Montagnier, L., & Charneau, P. (2000) · Cell · doi:10.1016/s0092-8674(00)80828-4 · Cited by 893 · see citing works

Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD

Nutt, J. G., Burchiel, K. J., Comella, C., Jankovic, J. J., Lang, A. E., Laws, E. R., Lozano, A. M., Penn, R. D., Simpson, R. K., Stacy, M. A., et al. (2003) · Neurology · doi:10.1212/wnl.60.1.69 · Cited by 816 · see citing works

Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus

Lewis, P. F., & Emerman, M. (1994) · Journal of Virology · doi:10.1128/jvi.68.1.510-516.1994 · Cited by 797 · see citing works

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

McCarty, D., Monahan, P., & Samulski, R. (2001) · Gene Therapy · doi:10.1038/sj.gt.3301514 · Cited by 735 · see citing works

Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector

Blömer, U., Naldini, L., Kafri, T., Trono, D., Verma, I. M., & Gage, F. H. (1997) · Journal of Virology · doi:10.1128/jvi.71.9.6641-6649.1997 · Cited by 725 · see citing works

Transplantation of Umbilical-Cord Blood in Babies with Infantile Krabbe's Disease

Escolar, M. L., Poe, M. D., Provenzale, J. M., Richards, K. C., Allison, J., Wood, S. J., Wenger, D. A., Pietryga, D. W., Wall, D. A., Champagne, M. A., et al. (2005) · New England Journal of Medicine · doi:10.1056/nejmoa042604 · Cited by 703 · see citing works

Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration

Montini, E., Cesana, D., Schmidt, M., Sanvito, F., Ponzoni, M., Bartholomae, C. C., Sergi, L. S., Benedicenti, F., Ambrosi, A., Serio, C. D., et al. (2006) · Nature Biotechnology · doi:10.1038/nbt1216 · Cited by 699 · see citing works

Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial

Marks, W. J., Bartus, R. T., Siffert, J. O., Davis, C. S., Lozano, A. M., Boulis, N. M., Vitek, J. L., Stacy, M. A., Turner, D. A., Verhagen, L., et al. (2010) · The Lancet Neurology · doi:10.1016/s1474-4422(10)70254-4 · Cited by 685 · see citing works

AAV2-GAD gene therapy for advanced Parkinson’s disease: a double-blind, sham-surgery controlled, randomised trial

LeWitt, P. A., Rezai, A. R., Leehey, M. A., Ojemann, S. G., Flaherty, A. W., Eskandar, E. N., Kostyk, S. K., Thomas, K., Sarkar, A., Siddiqui, M. S., et al. (2011) · The Lancet Neurology · doi:10.1016/s1474-4422(11)70039-4 · Cited by 681 · see citing works

Intricate Regulation of Tyrosine Hydroxylase Activity and Gene Expression

Kumer, S. C., & Vrana, K. E. (1996) · Journal of Neurochemistry · doi:10.1046/j.1471-4159.1996.67020443.x · Cited by 680 · see citing works

Immune responses to gene therapy vectors: influence on vector function and effector mechanisms

Bessis, N., Garcia-Cozar, F. J., & Boissier, M. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302364 · Cited by 636 · see citing works

Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2–neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial

Marks, W. J., Ostrem, J. L., Verhagen, L., Starr, P. A., Larson, P. S., Bakay, R. A., Taylor, R., Cahn-Weiner, D. A., Stoessl, A. J., Olanow, C. W., et al. (2008) · The Lancet Neurology · doi:10.1016/s1474-4422(08)70065-6 · Cited by 596 · see citing works

VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model

Azzouz, M., Ralph, G. S., Storkebaum, E., Walmsley, L. E., Mitrophanous, K. A., Kingsman, S. M., Carmeliet, P., & Mazarakis, N. D. (2004) · Nature · doi:10.1038/nature02544 · Cited by 588 · see citing works

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

Montini, E., Cesana, D., Schmidt, M., Sanvito, F., Bartholomae, C. C., Ranzani, M., Benedicenti, F., Sergi, L. S., Ambrosi, A., Ponzoni, M., et al. (2009) · Journal of Clinical Investigation · doi:10.1172/jci37630 · Cited by 571 · see citing works

Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

McCarty, D. M., Fu, H., Monahan, P. E., Toulson, C. E., Naik, P., & Samulski, R. J. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302134 · Cited by 547 · see citing works

Altering the Tropism of Lentiviral Vectors through Pseudotyping

Cronin, J., Zhang, X.-Y., & Reiser, J. (2005) · Current Gene Therapy · doi:10.2174/1566523054546224 · Cited by 540 · see citing works

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

Boztug, K., Schmidt, M., Schwarzer, A., Banerjee, P. P., Díez, I. A., Dewey, R. A., Böhm, M., Nowrouzi, A., Ball, C. R., Glimm, H., et al. (2010) · New England Journal of Medicine · doi:10.1056/nejmoa1003548 · Cited by 532 · see citing works

Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons

Duque, S., Joussemet, B., Riviere, C., Marais, T., Dubreil, L., Douar, A.-M., Fyfe, J., Moullier, P., Colle, M.-A., & Barkats, M. (2009) · Molecular Therapy · doi:10.1038/mt.2009.71 · Cited by 511 · see citing works

Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model

Ralph, G. S., Radcliffe, P. A., Day, D. M., Carthy, J. M., Leroux, M. A., Lee, D. C. P., Wong, L., Bilsland, L. G., Greensmith, L., Kingsman, S. M., et al. (2005) · Nature Medicine · doi:10.1038/nm1205 · Cited by 507 · see citing works

Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates

Gray, S. J., Matagne, V., Bachaboina, L., Yadav, S., Ojeda, S. R., & Samulski, R. J. (2011) · Molecular Therapy · doi:10.1038/mt.2011.72 · Cited by 501 · see citing works

New recombinant serotypes of AAV vectors

Gao, G., Vandenberghe, L., & Wilson, J. (2005) · Current Gene Therapy · doi:10.2174/1566523054065057 · Cited by 487 · see citing works

Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS

Raoul, C., Abbas‐Terki, T., Bensadoun, J., Guillot, S., Haase, G., Szulc, J., Henderson, C. E., & Aebischer, P. (2005) · Nature Medicine · doi:10.1038/nm1207 · Cited by 464 · see citing works

Potentiation of Cationic Liposome-Mediated Gene Delivery by Polycations

Gao, X., & Huang, L. (1996) · Biochemistry · doi:10.1021/bi952436a · Cited by 461 · see citing works

Effective gene therapy with nonintegrating lentiviral vectors

Yáñez-Muñoz, R. J., Balaggan, K. S., MacNeil, A., Howe, S. J., Schmidt, M., Smith, A. J., Buch, P., MacLaren, R. E., Anderson, P. N., Barker, S. E., et al. (2006) · Nature Medicine · doi:10.1038/nm1365 · Cited by 460 · see citing works

Integration of Adeno-Associated Virus (AAV) and Recombinant AAV Vectors

McCarty, D. M., Young, S. M., & Samulski, R. J. (2004) · Annual Review of Genetics · doi:10.1146/annurev.genet.37.110801.143717 · Cited by 450 · see citing works

Safety and tolerability of putaminal <i>AADC</i> gene therapy for Parkinson disease

Christine, C. W., Starr, P. A., Larson, P. S., Eberling, J. L., Jagust, W. J., Hawkins, R. A., VanBrocklin, H. F., Wright, J. F., Bankiewicz, K. S., & Aminoff, M. J. (2009) · Neurology · doi:10.1212/wnl.0b013e3181c29356 · Cited by 445 · see citing works

The IN protein of Moloney murine leukemia virus processes the viral DNA ends and accomplishes their integration in vitro

Craigie, R., Fujiwara, T., & Bushman, F. (1990) · Cell · doi:10.1016/0092-8674(90)90126-y · Cited by 409 · see citing works

Convection-Enhanced Delivery of AAV Vector in Parkinsonian Monkeys; In Vivo Detection of Gene Expression and Restoration of Dopaminergic Function Using Pro-drug Approach

Bankiewicz, K. S., Eberling, J. L., Kohutnicka, M., Jagust, W. J., Pivirotto, P., Bringas, J., Cunningham, J., Budinger, T. F., & Harvey-White, J. D. (2000) · Experimental Neurology · doi:10.1006/exnr.2000.7408 · Cited by 406 · see citing works

Aspartoacylase deficiency and N‐acetylaspartic aciduria in patients with canavan disease

Matalon, R. K., Michals, K., Sebesta, D. G., Deanching, M., Gashkoff, P., Casanova, J., Optiz, J. M., & Reynolds, J. F. (1988) · American Journal of Medical Genetics · doi:10.1002/ajmg.1320290234 · Cited by 405 · see citing works

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA

Worgall, S., Sondhi, D., Hackett, N. R., Kosofsky, B., Kekatpure, M. V., Neyzi, N., Dyke, J. P., Ballon, D., Heier, L., Greenwald, B. M., et al. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.022 · Cited by 402 · see citing works

The rev gene product of the human immunodeficiency virus affects envelope-specific RNA localization

Emerman, M., Vazeux, R., & Peden, K. (1989) · Cell · doi:10.1016/0092-8674(89)90053-6 · Cited by 396 · see citing works

Insertional Transformation of Hematopoietic Cells by Self-inactivating Lentiviral and Gammaretroviral Vectors

Modlich, U., Navarro, S., Zychlinski, D., Maetzig, T., Knoess, S., Brugman, M. H., Schambach, A., Charrier, S., Galy, A., Thrasher, A. J., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.179 · Cited by 387 · see citing works

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

Gray, S. J., Nagabhushan Kalburgi, S., McCown, T. J., & Jude Samulski, R. (2013) · Gene Therapy · doi:10.1038/gt.2012.101 · Cited by 377 · see citing works

Gene delivery by lentivirus vectors

Cockrell, A. S., & Kafri, T. (2007) · Molecular Biotechnology · doi:10.1007/s12033-007-0010-8 · Cited by 358 · see citing works

The rev (trs/art) protein of human immunodeficiency virus type 1 affects viral mRNA and protein expression via a cis-acting sequence in the env region

Hadzopoulou-Cladaras, M., Felber, B. K., Cladaras, C., Athanassopoulos, A., Tse, A., & Pavlakis, G. N. (1989) · Journal of Virology · doi:10.1128/jvi.63.3.1265-1274.1989 · Cited by 351 · see citing works

A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson’s disease

Muramatsu, S.-I., Fujimoto, K.-I., Kato, S., Mizukami, H., Asari, S., Ikeguchi, K., Kawakami, T., Urabe, M., Kume, A., Sato, T., et al. (2010) · Molecular Therapy · doi:10.1038/mt.2010.135 · Cited by 344 · see citing works

Subthalamic GAD Gene Therapy in a Parkinson's Disease Rat Model

Luo, J., Kaplitt, M. G., Fitzsimons, H. L., Zuzga, D. S., Liu, Y., Oshinsky, M. L., & During, M. J. (2002) · Science · doi:10.1126/science.1074549 · Cited by 325 · see citing works

Molecular cloning and complete nucleotide sequence of the attenuated rabies virus SAD B19

Conzelmann, K., Cox, J. H., Schneider, L. G., & Thiel, H. (1990) · Virology · doi:10.1016/0042-6822(90)90433-r · Cited by 324 · see citing works

Immune Responses to AAV in Clinical Trials

Mingozzi, F., & A. High, K. (2011) · Current Gene Therapy · doi:10.2174/156652311796150354 · Cited by 324 · see citing works

Lentiviral vector delivery of parkin prevents dopaminergic degeneration in an α-synuclein rat model of Parkinson's disease

Bianco, C. L., Schneider, B. L., Bauer, M., Sajadi, A., Brice, A., Iwatsubo, T., & Aebischer, P. (2004) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0405313101 · Cited by 322 · see citing works

High-Titer Human Immunodeficiency Virus Type 1-Based Vector Systems for Gene Delivery into Nondividing Cells

Mochizuki, H., Schwartz, J. P., Tanaka, K., Brady, R. O., & Reiser, J. (1998) · Journal of Virology · doi:10.1128/jvi.72.11.8873-8883.1998 · Cited by 308 · see citing works

Long-term follow-up after gene therapy for canavan disease

Leone, P., Shera, D., McPhee, S. W. J., Francis, J. S., Kolodny, E. H., Bilaniuk, L. T., Wang, D.-J., Assadi, M., Goldfarb, O., Goldman, H. W., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3003454 · Cited by 307 · see citing works

Pseudotype formation of murine leukemia virus with the G protein of vesicular stomatitis virus

Emi, N., Friedmann, T., & Yee, J. K. (1991) · Journal of Virology · doi:10.1128/jvi.65.3.1202-1207.1991 · Cited by 302 · see citing works

Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector.

Gaspar, H., Parsley, K., Howe, S., King, D., Gilmour, K. C., Sinclair, J., Brouns, G., Schmidt, M., von Kalle, C., Barington, T., et al. (2004) · The Lancet · doi:10.1016/s0140-6736(04)17590-9 · Cited by 300 · see citing works

Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

Samaranch, L., Salegio, E. A., San Sebastian, W., Kells, A. P., Foust, K. D., Bringas, J. R., Lamarre, C., Forsayeth, J., Kaspar, B. K., & Bankiewicz, K. S. (2012) · Human Gene Therapy · doi:10.1089/hum.2011.200 · Cited by 299 · see citing works

Long-Term Evaluation of a Phase 1 Study of AADC Gene Therapy for Parkinson's Disease

Mittermeyer, G., Christine, C. W., Rosenbluth, K. H., Baker, S. L., Starr, P. A., Larson, P., Kaplan, P. L., Forsayeth, J., Aminoff, M. J., & Bankiewicz, K. S. (2012) · Human Gene Therapy · doi:10.1089/hum.2011.220 · Cited by 297 · see citing works

Distinct Genomic Integration of MLV and SIV Vectors in Primate Hematopoietic Stem and Progenitor Cells

Hematti, P., Hong, B., Ferguson, C., Adler, R. L., Hanawa, H., Sellers, S., Holt, I., Eckfeldt, C. E., Sharma, Y., Schmidt, M., et al. (2004) · PLoS Biology · doi:10.1371/journal.pbio.0020423 · Cited by 291 · see citing works

Gene therapy for lysosomal storage diseases

Sands, M. S., & Davidson, B. L. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.01.006 · Cited by 285 · see citing works

X-linked adrenoleukodystrophy: Clinical, metabolic, genetic and pathophysiological aspects

Kemp, S., Berger, J., & Aubourg, P. (2012) · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease · doi:10.1016/j.bbadis.2012.03.012 · Cited by 283 · see citing works

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

Biffi, A., Palma, M. D., Quattrini, A., Carro, U. D., Amadio, S., Visigalli, I., Sessa, M., Fasano, S., Brambilla, R., Marchesini, S., et al. (2004) · Journal of Clinical Investigation · doi:10.1172/jci200419205 · Cited by 280 · see citing works

A Packaging Cell Line for Lentivirus Vectors

Kafri, T., van Praag, H., Ouyang, L., Gage, F. H., & Verma, I. (1999) · Journal of Virology · doi:10.1128/jvi.73.1.576-584.1999 · Cited by 276 · see citing works

Adeno-associated virus: from defective virus to effective vector

Gonçalves, M. A. F. V. (2005) · Virology Journal · doi:10.1186/1743-422x-2-43 · Cited by 266 · see citing works

Genetic Fate of Recombinant Adeno-Associated Virus Vector Genomes in Muscle

Schnepp, B. C., Clark, K. R., Klemanski, D. L., Pacak, C. A., & Johnson, P. R. (2003) · Journal of Virology · doi:10.1128/jvi.77.6.3495-3504.2003 · Cited by 262 · see citing works

Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human <i>SGSH</i> and <i>SUMF1</i> cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/II Trial

Tardieu, M., Zérah, M., Husson, B., de Bournonville, S., Deiva, K., Adamsbaum, C., Vincent, F., Hocquemiller, M., Broissand, C., Furlan, V., et al. (2014) · Human Gene Therapy · doi:10.1089/hum.2013.238 · Cited by 261 · see citing works

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

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Gene Therapy of Canavan Disease: AAV-2 Vector for Neurosurgical Delivery of Aspartoacylase Gene ( <i>ASPA</i> ) to the Human Brain

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