Gene therapy clinical trials worldwide to 2017: An update

Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors

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A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity

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Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans

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Multiplex Genome Engineering Using CRISPR/Cas Systems

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RNA-Guided Human Genome Engineering via Cas9

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Axicabtagene Ciloleucel CAR T-Cell Therapy in Refractory Large B-Cell Lymphoma

Neelapu, S. S., Locke, F. L., Bartlett, N. L., Lekakis, L. J., Miklos, D. B., Jacobson, C. A., Braunschweig, I., Oluwole, O. O., Siddiqi, T., Lin, Y., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1707447 · Cited by 6,030

Development and Applications of CRISPR-Cas9 for Genome Engineering

Hsu, P. D., Lander, E. S., & Zhang, F. (2014) · Cell · doi:10.1016/j.cell.2014.05.010 · Cited by 5,762

Chimeric Antigen Receptor T Cells for Sustained Remissions in Leukemia

Maude, S. L., Frey, N., Shaw, P. A., Aplenc, R., Barrett, D. M., Bunin, N. J., Chew, A., Gonzalez, V. E., Zheng, Z., Lacey, S. F., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1407222 · Cited by 5,413

Chimeric antigen receptor–modified T cells in chronic lymphoid leukemia

Porter, D. L., Levine, B. L., Kalos, M., Bagg, A., & June, C. H. (2011) · New England Journal of Medicine · doi:10.1056/nejmoa1103849 · Cited by 3,565

Chimeric Antigen Receptor–Modified T Cells for Acute Lymphoid Leukemia

Grupp, S. A., Kalos, M., Barrett, D., Aplenc, R., Porter, D. L., Rheingold, S. R., Teachey, D. T., Chew, A., Hauck, B., Wright, J. F., et al. (2013) · New England Journal of Medicine · doi:10.1056/nejmoa1215134 · Cited by 3,509

Non-viral vectors for gene-based therapy

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Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA

Warren, L., Manos, P. D., Ahfeldt, T., Loh, Y.-H., Li, H., Lau, F., Ebina, W., Mandal, P. K., Smith, Z. D., Meissner, A., et al. (2010) · Cell Stem Cell · doi:10.1016/j.stem.2010.08.012 · Cited by 2,662

Cancer regression in patients after transfer of genetically engineered lymphocytes

Morgan, R. A., Dudley, M. E., Wunderlich, J. R., Hughes, M. S., Yang, J. C., Sherry, R. M., Royal, R. E., Topalian, S. L., Kammula, U. S., Restifo, N. P., et al. (2006) · Science · doi:10.1126/science.1129003 · Cited by 2,603

Efficacy and Toxicity Management of 19-28z CAR T Cell Therapy in B Cell Acute Lymphoblastic Leukemia

Davila, M. L., Rivière, I., Wang, X., Bartido, S. M., Park, J. H., Curran, K. J., Chung, S. S., Stefanski, J., Bórquez-Ojeda, O., Olszewska, M., et al. (2014) · Science Translational Medicine · doi:10.1126/scitranslmed.3008226 · Cited by 2,514

Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors

Boch, J., Scholze, H., Schornack, S., Landgraf, A., Hahn, S., Kay, S., Lahaye, T., Nickstadt, A., & Bonas, U. (2009) · Science · doi:10.1126/science.1178811 · Cited by 2,470

Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product

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T Cells with Chimeric Antigen Receptors Have Potent Antitumor Effects and Can Establish Memory in Patients with Advanced Leukemia

Kalos, M. D., Levine, B. L., Porter, D. L., Katz, S. I., Grupp, S. A., Bagg, A., & June, C. H. (2011) · Science Translational Medicine · doi:10.1126/scitranslmed.3002842 · Cited by 2,417

Human induced pluripotent stem cells free of vector and transgene sequences

Yu, J., Hu, K., Smuga-Otto, K., Tian, S., Stewart, R., Slukvin, I. I., & Thomson, J. A. (2009) · Science · doi:10.1126/science.1172482 · Cited by 2,386

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Mendell, J. R., Al-Zaidy, S., Shell, R., Arnold, W. D., Rodino-Klapac, L. R., Prior, T. W., Lowes, L., Alfano, L., Berry, K., Church, K., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1706198 · Cited by 2,381

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Finkel, R. S., Mercuri, E., Darras, B. T., Connolly, A. M., Kuntz, N. L., Kirschner, J., Chiriboga, C. A., Saito, K., Servais, L., Tizzano, E., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1702752 · Cited by 2,337

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

CD19-targeted T cells rapidly induce molecular remissions in adults with chemotherapy-refractory acute lymphoblastic leukemia

Brentjens, R. J., Davila, M. L., Riviere, I., Park, J., Wang, X., Cowell, L. G., Bartido, S., Stefanski, J., Taylor, C., Olszewska, M., et al. (2013) · Science Translational Medicine · doi:10.1126/scitranslmed.3005930 · Cited by 2,063

Cancer Immunotherapy

Couzin-Frankel, J. (2013) · Science · doi:10.1126/science.342.6165.1432 · Cited by 2,051

A Simple Cipher Governs DNA Recognition by TAL Effectors

Moscou, M., & Bogdanove, A. J. (2009) · Science · doi:10.1126/science.1178817 · Cited by 2,050

Altering the Genome by Homologous Recombination

Capecchi, M. R. (1989) · Science · doi:10.1126/science.2660260 · Cited by 2,037

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

Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain.

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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

A single dose of peripherally infused EGFRvIII-directed CAR T cells mediates antigen loss and induces adaptive resistance in patients with recurrent glioblastoma

O’Rourke, D. M., Nasrallah, M. P., Desai, A. S., Melenhorst, J. J., Mansfield, K. G., Morrissette, J. J. D., Martinez‐Lage, M., Brem, S. S., Maloney, E., Shen, A., et al. (2017) · Science Translational Medicine · doi:10.1126/scitranslmed.aaa0984 · Cited by 1,847

Regression of Glioblastoma after Chimeric Antigen Receptor T-Cell Therapy

Brown, C. E., Alizadeh, D., Starr, R., Weng, L., Wagner, J. R., Naranjo, A., Ostberg, J. R., Blanchard, M. S., Kilpatrick, J., Simpson, J., et al. (2016) · New England Journal of Medicine · doi:10.1056/nejmoa1610497 · Cited by 1,842

Rational design of cationic lipids for siRNA delivery

Semple, S. C., Akinc, A., Chen, J., Sandhu, A. P., Mui, B. L., Cho, C. K., Sah, D. W. Y., Stebbing, D., Crosley, E. J., Yaworski, E., et al. (2010) · Nature Biotechnology · doi:10.1038/nbt.1602 · Cited by 1,822 · cited 1×

Generation of Human Induced Pluripotent Stem Cells by Direct Delivery of Reprogramming Proteins

Kim, D., Kim, C.-H., Moon, J.-I., Chung, Y.-G., Chang, M.-Y., Han, B.-S., Ko, S., Yang, E., Cha, K. Y., Lanza, R., et al. (2009) · Cell stem cell · doi:10.1016/j.stem.2009.05.005 · Cited by 1,814

Tumor Regression in Patients With Metastatic Synovial Cell Sarcoma and Melanoma Using Genetically Engineered Lymphocytes Reactive With NY-ESO-1

Robbins, P. F., Morgan, R. A., Feldman, S. A., Yang, J. C.-H., Sherry, R. M., Dudley, M. E., Wunderlich, J. R., Nahvi, A. V., Helman, L. J., Mackall, C. L., et al. (2011) · Journal of Clinical Oncology · doi:10.1200/jco.2010.32.2537 · Cited by 1,635

Autologous Induced Stem-Cell–Derived Retinal Cells for Macular Degeneration

Mandai, M., Watanabe, A., Kurimoto, Y., Hirami, Y., Morinaga, C., Daimon, T., Fujihara, M., Akimaru, H., Sakai, N., Shibata, Y., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1608368 · Cited by 1,570

Induced pluripotent stem cell technology: a decade of progress.

Shi, Y., Inoue, H., Wu, J. C., & Yamanaka, S. (2017) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2016.245 · Cited by 1,525

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 · cited 2×

Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics

Kay, M., Glorioso, J., & Naldini, L. (2001) · Nature Medicine · doi:10.1038/83324 · Cited by 1,444

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

Tebas, P., Stein, D., Tang, W. W., Frank, I., Wang, S. Q., Lee, G., Spratt, S. K., Surosky, R. T., Giedlin, M. A., Nichol, G., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1300662 · Cited by 1,357

Gene Transfer into Humans — Immunotherapy of Patients with Advanced Melanoma, Using Tumor-Infiltrating Lymphocytes Modified by Retroviral Gene Transduction

Rosenberg, S. A., Aebersold, P. M., Cornetta, K. G., Kasid, A., Morgan, R. A., Moen, R. C., Karson, E. M., Lotze, M. T., Yang, J. C.-H., Topalian, S. L., et al. (1990) · New England Journal of Medicine · doi:10.1056/nejm199008303230904 · Cited by 1,343

Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies

Schwartz, S. D., Regillo, C. D., Lam, B. L., Eliott, D., Rosenfeld, P. J., Gregori, N. Z., Hubschman, J.-P., Davis, J. L., Heilwell, G., Spirn, M., et al. (2015) · The Lancet · doi:10.1016/s0140-6736(14)61376-3 · Cited by 1,327

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

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Nathwani, A. C., Reiss, U. M., Tuddenham, E. G. D., Rosales, C., Chowdary, P., McIntosh, J., Della Peruta, M., Lheriteau, E., Patel, N., Raj, D., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1407309 · Cited by 1,273

Lipid Nanoparticle Systems for Enabling Gene Therapies

Cullis, P. R., & Hope, M. J. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.03.013 · Cited by 1,208

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 1,187

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

CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes

Liang, P., Xu, Y., Zhang, X., Ding, C., Huang, R., Zhang, Z., Lv, J., Xie, X., Chen, Y., Li, Y., et al. (2015) · Protein & Cell · doi:10.1007/s13238-015-0153-5 · Cited by 1,135

Correction of a pathogenic gene mutation in human embryos

Ma, H., Marti-Gutierrez, N., Park, S.-W., Wu, J., Lee, Y., Suzuki, K., Koski, A., Ji, D., Hayama, T., Ahmed, R., et al. (2017) · Nature · doi:10.1038/nature23305 · Cited by 992

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

Yin, H., Xue, W., Chen, S., Bogorad, R. L., Benedetti, E., Grompe, M., Koteliansky, V., Sharp, P. A., Jacks, T., & Anderson, D. G. (2014) · Nature Biotechnology · doi:10.1038/nbt.2884 · Cited by 951

Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells

Qasim, W., Zhan, H., Samarasinghe, S., Adams, S., Amrolia, P., Stafford, S., Butler, K., Rivat, C., Wright, G., Somana, K., et al. (2017) · Science Translational Medicine · doi:10.1126/scitranslmed.aaj2013 · Cited by 902

HER2-Specific Chimeric Antigen Receptor–Modified Virus-Specific T Cells for Progressive Glioblastoma

Ahmed, N. M., Brawley, V. S., Hegde, M. G., Bielamowicz, K., Kalra, M., Landi, D. B., Robertson, C. S., Gray, T., Diouf, O., Wakefield, A., et al. (2017) · JAMA Oncology · doi:10.1001/jamaoncol.2017.0184 · Cited by 884

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Çırak, S., Arechavala‐Gomeza, V., Guglieri, M., Feng, L., Torelli, S., Anthony, K., Abbs, S. J., Garralda, M. E., Bourke, J. P., Wells, D. J., et al. (2011) · The Lancet · doi:10.1016/s0140-6736(11)60756-3 · Cited by 879

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

Maguire, A. M., High, K. A., Auricchio, A., Wright, J. F., Pierce, E. A., Testa, F., Mingozzi, F., Bennicelli, J. L., Ying, G.-S., Rossi, S., et al. (2009) · The Lancet · doi:10.1016/s0140-6736(09)61836-5 · Cited by 865

Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA − Immunodeficient Patients

Bordignon, C., Notarangelo, L. D., Nobili, N., Ferrari, G., Casorati, G., Panina‐Bordignon, P., Mazzolari, E., Maggioni, D., Rossi, C., Servida, P., et al. (1995) · Science · doi:10.1126/science.270.5235.470 · Cited by 830

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

George, L. A., Sullivan, S. K., Giermasz, A., Rasko, J. E. J., Samelson-Jones, B. J., Ducore, J., Cuker, A., Sullivan, L. M., Majumdar, S., Teitel, J., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1708538 · Cited by 762

Safety, efficacy, and immunogenicity of VGX-3100, a therapeutic synthetic DNA vaccine targeting human papillomavirus 16 and 18 E6 and E7 proteins for cervical intraepithelial neoplasia 2/3: a randomised, double-blind, placebo-controlled phase 2b trial

Trimble, C. L., Morrow, M. P., Kraynyak, K. A., Shen, X., Dallas, M. J., Yan, J., Edwards, L., Parker, R. L., Denny, L., Giffear, M., et al. (2015) · The Lancet · doi:10.1016/s0140-6736(15)00239-1 · Cited by 754

A prudent path forward for genomic engineering and germline gene modification

Baltimore, D., Berg, P., Botchan, M., Carroll, D., Charo, R. A., Church, G., Corn, J. E., Daley, G. Q., Doudna, J. A., Fenner, M., et al. (2015) · Science · doi:10.1126/science.aab1028 · Cited by 733

First-in-Humans Trial of an RNA Interference Therapeutic Targeting VEGF and KSP in Cancer Patients with Liver Involvement

Tabernero, J. M., Shapiro, G. I., LoRusso, P. M., Cervantes, A. M. R., Schwartz, G. K., Weiss, G. J., Paz-Ares, L. G., Cho, D. C., Infante, J. R., Alsina, M., et al. (2013) · Cancer Discovery · doi:10.1158/2159-8290.cd-12-0429 · Cited by 717

Inactivation of porcine endogenous retrovirus in pigs using CRISPR-Cas9

Niu, D., Wei, H.-J., Lin, L., George, H., Wang, T., Lee, I.-H., Zhao, H.-Y., Wang, Y., Kan, Y., Shrock, E., et al. (2017) · Science · doi:10.1126/science.aan4187 · Cited by 717

Gene therapy in a patient with sickle cell disease

Ribeil, J.-A., Hacein-Bey-Abina, S., Payen, E., Magnani, A., Semeraro, M., Magrin, E., Caccavelli, L., Neven, B., Bourget, P., El Nemer, W., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1609677 · Cited by 696

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

Hacein‐Bey‐Abina, S., Hauer, J., Lim, A., Pïcard, C., Wang, G. P., Berry, C. C., Martinache, C., Rieux‐Laucat, F., Latour, S., Belohradsky, B. H., et al. (2010) · New England Journal of Medicine · doi:10.1056/nejmoa1000164 · Cited by 654

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

Kinali, M., Arechavala‐Gomeza, V., Feng, L., Çırak, S., Hunt, D. M., Adkin, C. F., Guglieri, M., Ashton, E., Abbs, S. J., Nihoyannopoulos, P., et al. (2009) · The Lancet Neurology · doi:10.1016/s1474-4422(09)70211-x · Cited by 653

Clinical manufacturing of CAR T cells: foundation of a promising therapy

Wang, X., & Rivière, I. (2016) · Molecular Therapy - Oncolytics · doi:10.1038/mto.2016.15 · Cited by 637

GENE THERAPY: Twenty-First Century Medicine

Verma, I. M., & Weitzman, M. D. (2005) · Annual Review of Biochemistry · doi:10.1146/annurev.biochem.74.050304.091637 · Cited by 630

Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles

Wang, M., Zuris, J. A., Meng, F., Rees, H., Sun, S., Deng, P., Han, Y., Gao, X., Pouli, D., Wu, Q., et al. (2016) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1520244113 · Cited by 624

Nucleic Acid Therapeutics Using Polyplexes: A Journey of 50 Years (and Beyond)

Lächelt, U., & Wagner, E. (2015) · Chemical Reviews · doi:10.1021/cr5006793 · Cited by 607

Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy

Eichler, F., Duncan, C., Musolino, P. L., Orchard, P. J., De Oliveira, S., Thrasher, A. J., Armant, M., Dansereau, C., Lund, T. C., Miller, W. P., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1700554 · Cited by 604

Gene therapy clinical trials worldwide to 2007—an update

Edelstein, M. L., Abedi, M. R., & Wixon, J. (2007) · The Journal of Gene Medicine · doi:10.1002/jgm.1100 · Cited by 595

Gene therapy for Wiskott-Aldrich syndrome–long-term efficacy and genotoxicity

Braun, C. J., Boztug, K., Paruzynski, A., Witzel, M., Schwarzer, A., Rothe, M., Modlich, U., Beier, R., Göhring, G., Steinemann, D., et al. (2014) · Science Translational Medicine · doi:10.1126/scitranslmed.3007280 · Cited by 562

Gene therapy: trials and tribulations

Somia, N., & Verma, I. M. (2000) · Nature Reviews Genetics · doi:10.1038/35038533 · Cited by 554

Toward controlling gene expression at will: Specific regulation of the erbB - 2 / HER - 2 promoter by using polydactyl zinc finger proteins constructed from modular building blocks

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Adenoviral gene delivery can reprogram human fibroblasts to induced pluripotent stem cells

Zhou, W., & Freed, C. R. (2009) · Stem Cells · doi:10.1002/stem.201 · Cited by 538

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

Don’t edit the human germ line

Lanphier, E., Urnov, F., Haecker, S. E., Werner, M., & Smolenski, J. (2015) · Nature · doi:10.1038/519410a · Cited by 530

Phase I Escalating-Dose Trial of CAR-T Therapy Targeting CEA+ Metastatic Colorectal Cancers

Zhang, C., Wang, Z., Yang, Z., Wang, M., Li, S., Li, Y., Zhang, R., Xiong, Z., Wei, Z., Shen, J., et al. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.03.010 · Cited by 463

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

Bennett, J., Wellman, J., Marshall, K. A., McCague, S., Ashtari, M., DiStefano-Pappas, J., Elci, O. U., Chung, D. C., Sun, J., Wright, J. F., et al. (2016) · The Lancet · doi:10.1016/s0140-6736(16)30371-3 · Cited by 461

Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial

Alton, E. W. F. W., Armstrong, D. K., Ashby, D., Bayfield, K. J., Bilton, D., Bloomfield, E. V., Boyd, A. C., Brand, J., Buchan, R., Calcedo, R., et al. (2015) · The Lancet Respiratory Medicine · doi:10.1016/s2213-2600(15)00245-3 · Cited by 428

Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome

Hacein‐Bey‐Abina, S., Gaspar, H. B., Blondeau, J., Caccavelli, L., Charrier, S., Buckland, K. F., Pïcard, C., Six, E. M., Himoudi, N., Gilmour, K. C., et al. (2015) · JAMA · doi:10.1001/jama.2015.3253 · Cited by 391

Gene therapy clinical trials worldwide 1989–2004—an overview

Edelstein, M. L., Abedi, M. R., Wixon, J., & Edelstein, R. M. (2004) · Journal of Gene Medicine · doi:10.1002/jgm.619 · Cited by 371

FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

Aartsma-Rus, A. M., & Krieg, A. Μ. (2016) · Nucleic Acid Therapeutics · doi:10.1089/nat.2016.0657 · Cited by 364

CRISPR gene-editing tested in a person for the first time

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Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes

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Summary

Med, J. G., Edelstein, M. L., Abedi, M. R., & Wixon, J. (2007)

Current Perspectives and Trends in Gene Therapy and Their Clinical Trials

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