The DOI matches its Crossref record; 146 of the 146 references it cites were found in a registry or an index.
Gene therapy clinical trials worldwide to 2017: An update
Abstract
To date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have been approved worldwide. Our database brings together global information on gene therapy clinical activity from trial databases, official agency sources, published literature, conference presentations and posters kindly provided to us by individual investigators or trial sponsors. This review presents our analysis of clinical trials that, to the best of our knowledge, have been or are being performed worldwide. As of our November 2017 update, we have entries on 2597 trials undertaken in 38 countries. We have analysed the geographical distribution of trials, the disease indications (or other reasons) for trials, the proportions to which different vector types are used, and the genes that have been transferred. Details of the analyses presented, and our searchable database are available via The Journal of Gene Medicine Gene Therapy Clinical Trials Worldwide website at: http://www.wiley.co.uk/genmed/clinical. We also provide an overview of the progress being made in gene therapy clinical trials around the world, and discuss key trends since the previous review, namely the use of chimeric antigen receptor T cells for the treatment of cancer and advancements in genome editing technologies, which have the potential to transform the field moving forward.
Overview
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
In the citing paper
“About 18.5% of clinical trials use this vector for gene therapy [57].”
From The Ocular Gene Delivery Landscape (2021) · cites this paper as [57]
In the citing paper
“Furthermore, some viral vectors (eg, Adenovirus vector, Herpesviral vector) are toxic and can be immunogenic.”
From Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges (2020) · cites this paper as [22]
In the citing paper
“The AV vector now represents 1 of the 3 major viral vector categories in the gene therapy toolbox, along with AAV and lenti/retrovirus vector systems.”
From Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges (2020) · cites this paper as [22]
Citations
Cites
Takahashi, K., & Yamanaka, S. (2006) · Cell · doi:10.1016/j.cell.2006.07.024 · Cited by 26,673
A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity
Jinek, M., Chylinski, K., Fonfara, I., Hauer, M., Doudna, J. A., & Charpentier, E. (2012) · Science · doi:10.1126/science.1225829 · Cited by 17,116
Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
Fire, A., Xu, S., Montgomery, M. K., Kostas, S. A., Driver, S. E., & Mello, C. C. (1998) · Nature · doi:10.1038/35888 · Cited by 15,429
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
RNA-Guided Human Genome Engineering via Cas9
Mali, P., Yang, L., Esvelt, K. M., Aach, J., Guell, M., DiCarlo, J. E., Norville, J. E., & Church, G. M. (2013) · Science · doi:10.1126/science.1232033 · Cited by 9,210
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
Flagged first, then by citations
Research works printed but not linked: 6
- Aiuti A, 2009, Ten years of gene therapy for primary immune deficiencies. Hematology 2009 – the Education Program of the American Society of Hematology, Education Program, 2009, 682
- GeorgeLA RagniMV Samelson‐JonesBJ et al.Preliminary results from a phase 1/2 dose escalation trial of an investigational AAV‐mediated gene therapy for hemophilia A. American Society of Hematology 59th Annual Meeting and Exposition. Online Conference Proceedings; 9 December2017.https://ash.confex.com/ash/2017/webprogram/Paper108679.html[30 January 2018].
- Constable IJ, 2016, Phase 2a randomized clinical trial: safety and post hoc analysis of subretinal rAAV.sFLT‐1 for wet age‐related macular degeneration. E, Biol Med, 14, 168
- MonahanP WalshCE PowellJS et al.Update on a phase 1/2 open‐label trial of BAX335 an adeno‐associated virus 8 (AAV8) vector‐based gene therapy program for hemophilia B. Proceedings of the XXV International Society on Thrombosis and Haemostasis Conference.North Carolina USA:Carrboro;2015;13(Suppl 2): LB010):87.
- Abeona Therapeutics.Abeona Therapeutics Receives Regulatory Approval to Initiate Clinical Trial in Australia with ABO‐102 Gene Therapy For Patients with MPS IIIA: Clinical Trial Notification (CTN) Enables Initiation at 3rd Global Site and Accelerates Enrollment in the ABO‐102 MPS IIIA Gene Therapy Program. Abeona Therapeutics: Press Release 9 May2017.https://globenewswire.com/news-release/2017/05/09/980972/0/en/Abeona-Therapeutics-Receives-Regulatory-Approval-to-Initiate-Clinical-Trial-in-Australia-with-ABO-102-Gene-Therapy-For-Patients-with-MPS-IIIA.html[3 April 2018].
- Amgen.FDA Approves IMLYGIC™ (Talimogene Laherparepvec) As First Oncolytic Viral Therapy In The US. AMGEN website: Press Release 27 October2015.http://www.amgen.com/media/news‐releases/2015/10/fda‐approves‐imlygic‐talimogene‐laherparepvec‐as‐first‐oncolytic‐viral‐therapy‐in‐the‐us/[30 January 2018].
Cited by
Sorted by citations
Items 501–600 of 903
Efficient inhibition of ovarian cancer by degradable nanoparticle-delivered survivin T34A gene
Luo, L., Du, T., Zhang, J., Zhao, W., Men, K., Gou, M., Cheng, H., Yang, Y., Wu, Y., & Wang, C. (2016) · International Journal of Nanomedicine · doi:10.2147/ijn.s93496 · Cited by 8
Bieńkowska, A., Ducher, M., Orzechowska, M., Słyk, Ż., Ciepiela, O., Jaworowski, J., & Małecki, M. (2019) · Experimental and Therapeutic Medicine · doi:10.3892/etm.2019.8112 · Cited by 8
Biomaterial-assisted gene therapy for translational approaches to treat musculoskeletal disorders
Venkatesan, J. K., Rey‐Rico, A., Meng, W., Cai, X., Pons, F., Lebeau, L., Migonney, V., Madry, H., & Cucchiarini, M. (2020) · Materials Today Advances · doi:10.1016/j.mtadv.2020.100126 · Cited by 9
Targeted gene delivery to the brain using CDX-modified chitosan nanoparticles
Sepasi, T., Bani, F., Rahbarghazi, R., Kalan, A. E., Sadeghi, M.-R., Alamolhoda, S. Z., Zarebkohan, A., Ghadiri, T., & Gao, H. (2022) · Bioimpacts · doi:10.34172/bi.2022.23876 · Cited by 8
Hu, D., Fumoto, S., Yoshikawa, N., Peng, J., Miyamoto, H., Tanaka, M., & Nishida, K. (2023) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2023.122881 · Cited by 8
Arabipour, I., Amani, J., Mirhosseini, S. A., & Salimian, J. (2019) · Gene · doi:10.1016/j.gene.2019.143968 · Cited by 9
Future Preventive Gene Therapy of Polygenic Diseases from a Population Genetics Perspective
Oliynyk, R. T. (2019) · International Journal of Molecular Sciences · doi:10.3390/ijms20205013 · Cited by 8
Nanoparticle‐Mediated Suicide Gene Therapy for Triple Negative Breast Cancer Treatment
Salvioni, L., Zuppone, S., Andreata, F., Monieri, M., Mazzucchelli, S., Carlo, C. D., Morelli, L., Cordiglieri, C., Donnici, L., Francesco, R. D., et al. (2020) · Advanced Therapeutics · doi:10.1002/adtp.202000007 · Cited by 8
Cationic lipid-based formulations for encapsulation and delivery of anti-EFG1 2′ OMethylRNA oligomer
Araújo, D., Gaspar, R., Mil‐Homens, D., Henriques, M., Silva, B. F. B., & Silva, S. M. (2022) · Medical Mycology · doi:10.1093/mmy/myac030 · Cited by 8
PEGylated Amine-Functionalized Poly(ε-caprolactone) for the Delivery of Plasmid DNA
Jafari, A., Rajabian, N., Zhang, G., Mohamed, M. A., Lei, P., Andreadis, S. T., Pfeifer, B. A., & Cheng, C. (2020) · Materials · doi:10.3390/ma13040898 · Cited by 8
Nanocarriers: A Tool for Effective Gene Delivery
Wadetwar, R. N., & Godbole, A. P. (2021) · doi:10.1002/9781119711698.ch8 · Cited by 8
(2024) · doi:10.1016/j.omton.2024.200805 · Cited by 8
Reshetnikov, V. V., Chirinskaite, A. V., Sopova, J. V., Ivanov, R. A., & Леонова, Е. И. (2022) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2022.903812 · Cited by 8
Yang, Y., Bian, L., Yuan, C., Xu, Y., Shao, H., Rao, J., Ge, S., Gong, J., Jiang, M., Zheng, X., et al. (2025) · Clinical and Translational Science · doi:10.1111/cts.70148 · Cited by 7
A novel miniaturized filamentous phagemid as a gene delivery vehicle to target mammalian cells
Wong, S. L., Jimenez, S., Pushparajah, D., Prakash, R., & Slavcev, R. A. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102571 · Cited by 7
Zhang, H., Li, Y., Li, J., Li, X., & Li, T. (2025) · Pharmaceutics · doi:10.3390/pharmaceutics17111405 · Cited by 8
Tahmasbi, L., Sedaghat, T., Motamedi, H., & Kooti, M. (2023) · Materials Advances · doi:10.1039/d3ma00066d · Cited by 7
Navigating the tumor microenvironment: mesenchymal stem cell-mediated delivery of anticancer agents
Waqar, M. A., Zaman, M., Khan, R., Rahman, M. S. U., & Majeed, I. (2024) · Journal of drug targeting · doi:10.1080/1061186x.2024.2347356 · Cited by 7
Mapfumo, P. P., Reichel, L. S., André, T., Hoeppener, S., Rudolph, K. L., & Traeger, A. (2024) · Biomacromolecules · doi:10.1021/acs.biomac.4c00007 · Cited by 8
Gene therapy for retinal diseases: From genetics to treatment
Khaparde, A., Mathias, G. P., Poornachandra, B., Thirumalesh, M. B., Shetty, R., & Ghosh, A. (2024) · Indian Journal of Ophthalmology · doi:10.4103/ijo.ijo_2902_23 · Cited by 8
Gay, L., Suwan, K., & Hajitou, A. (2024) · Nature Protocols · doi:10.1038/s41596-024-01040-9 · Cited by 7
A biofunctionalized viral delivery patch for spatially defined transfection
Kim, S., Yu, S. J., Kim, I., Choi, J., Choi, Y. H., Im, S. G., & Hwang, N. S.-Y. (2019) · Chemical Communications · doi:10.1039/c8cc09768b · Cited by 7
Concise review: Extracellular vesicles from mesenchymal stem cells as cellular therapy
Pham, P. V. (2017) · Biomedical Research and Therapy · doi:10.15419/bmrat.v4i08.287 · Cited by 8
Cho, M., Jung, K., Kim, S., Kim, I., Kim, M., Shin, M., Lee, H., Park, K. I., & Jang, J. (2019) · Gene Therapy · doi:10.1038/s41434-019-0057-8 · Cited by 7
Genome editing in the human liver: Progress and translational considerations
Ginn, S. L., Christina, S., & Alexander, I. E. (2021) · Progress in molecular biology and translational science · doi:10.1016/bs.pmbts.2021.01.030 · Cited by 7
Self-citationUnresolved Issues in RNA Therapeutics in Vascular Diseases With a Focus on Aneurysm Disease
Schellinger, I. N., Dannert, A., Mattern, K., Raaz, U., & Tsao, P. S. (2021) · Frontiers in Cardiovascular Medicine · doi:10.3389/fcvm.2021.571076 · Cited by 8
Cortijo-Gutierréz, M., Sánchez-Hernández, S., Tristán‐Manzano, M., Maldonado‐Pérez, N., López-Onieva, L., Real, P. J., Herrera, C., Marchal, J. A., Martín, F., & Benabdellah, K. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13081217 · Cited by 7
Dooms, M. M., Saesen, R., Steemans, I., Lansens, J., & Huys, I. (2022) · Frontiers in Pharmacology · doi:10.3389/fphar.2022.775217 · Cited by 7
Maternal diabetes negatively impacts fetal health
Corona, C. G., & Parchem, R. J. (2022) · Open Biology · doi:10.1098/rsob.220135 · Cited by 8
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future
Quillien, L., Buscail, L., & Cordelier, P. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.210 · Cited by 8
Gene therapy for pediatric genetic kidney diseases
Lu, Y., Song, Y., Sun, S., Zhang, L., & Chen, Y. (2023) · Pediatric Discovery · doi:10.1002/pdi3.16 · Cited by 8
Badr, K. R., Elmahdy, E. M., & Shaheen, M. N. F. (2024) · Water Science & Technology Water Supply · doi:10.2166/ws.2024.001 · Cited by 7
Kimura, S., & Harashima, H. (2024) · BioDrugs · doi:10.1007/s40259-024-00647-4 · Cited by 8
Bioengineering strategies for gene delivery
Shams, S., & Silva, E. A. (2019) · Elsevier eBooks · doi:10.1016/b978-0-12-816221-7.00004-5 · Cited by 8
Peptide modified polycations with pH triggered lytic activity for efficient gene delivery
Chen, X., Xu, K. L., Yu, J. X., Zhao, X., Zhang, Q., Zhang, Y., & Cheng, Y. (2020) · Biomaterials Science · doi:10.1039/d0bm01231a · Cited by 7
Leikas, A. J., Laham-Karam, N., Agtereek, E., Peltonen, H. M., Selander, T. A., Korpisalo, P., Holappa, L., Hartikainen, J. E. K., Heikura, T., & Ylä‐Herttuala, S. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.299 · Cited by 7
Shi, S., Li, F. R., Wu, L., Zhang, L., & Liu, L. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.214 · Cited by 7
Liposomes: An Emerging Approach for the Treatment of Cancer
Mishra, K., & Jain, A. K. (2021) · Current Pharmaceutical Design · doi:10.2174/1381612827666210406141449 · Cited by 7
Application of Genetic Engineering in Biotherapeutics Development
Schweickert, P. G., & Cheng, Z. (2019) · Journal of Pharmaceutical Innovation · doi:10.1007/s12247-019-09411-6 · Cited by 8
Tong, Y., Wan, W., Yang, H., Wang, Y., Wang, D., Liu, Y., You, B.-G., & Zhang, X. (2020) · doi:10.21203/rs.3.rs-20688/v1 · Cited by 7
Fischer, A., Ehrlich, A., Plotkin, Y., Ouyang, Y., Asulin, K., Ioannidis, K., Fan, C., Nahmias, Y., & Willner, I. (2023) · Angewandte Chemie · doi:10.1002/ange.202311590 · Cited by 6
Gene therapy for the heart: encapsulated viruses to the rescue
Deshetty, U. M., Sil, S., & Buch, S. J. (2024) · Extracellular Vesicles and Circulating Nucleic Acids · doi:10.20517/evcna.2023.70 · Cited by 7
VEGF—Virus Interactions: Pathogenic Mechanisms and Therapeutic Applications
Sánchez-Martínez, C., Grueso, E., Calvo-López, T., Martínez-Ortega, J., Ruiz, A., & Almendral, J. M. (2024) · Cells · doi:10.3390/cells13211815 · Cited by 6
Hemiacetal-linked pH-sensitive PEG-lipids for non-viral gene delivery
Coelho, F., Salonen, L. M., & Silva, B. F. B. (2022) · New Journal of Chemistry · doi:10.1039/d2nj02217f · Cited by 6
Prospective approaches to gene therapy computational modeling – spotlight on viral gene therapy
Choules, M. P., Bonate, P. L., Heo, N., & Weddell, J. C. (2023) · Journal of Pharmacokinetics and Pharmacodynamics · doi:10.1007/s10928-023-09889-1 · Cited by 7
A facile and scalable method to synthesize PEGylated PDMAEMA for gene delivery
Dou, J., Yu, S., & Zhang, Y. (2024) · Biopolymers · doi:10.1002/bip.23584 · Cited by 6
An Eye on Extracellular Vesicles: Trends and Clinical Translations in Vision Research
Dhodapkar, R. M., Jung, E. E., & Lee, S. Y. (2024) · Ophthalmology Science · doi:10.1016/j.xops.2024.100619 · Cited by 6
Zou, H., Tuhin, I. J., Monty, M. A., Luo, S., Shao, J., Yan, Z., & Yu, L. (2020) · Human Gene Therapy · doi:10.1089/hum.2019.169 · Cited by 6
Cao, H., Duan, R. C., & Hu, J. (2020) · Genes · doi:10.3390/genes11050565 · Cited by 6
Агатиева, Э. А., Ksembaev, S. S., Sokolov, M. E., Аршалуйсович, М. В., Gazizov, I. M., Tsyplakov, D. E., Шмаров, М. М., Леонидовна, Т. И., Naroditsky, B. S., Logunov, D. Y., et al. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13010058 · Cited by 6
Lomazzi, M., Franceschi, V., Bagnacani, V., Vezzoni, C. A., Donofrío, G., Casnati, A., & Sansone, F. (2021) · European Journal of Organic Chemistry · doi:10.1002/ejoc.202100338 · Cited by 6
Holland, L., He, Y., Guerrette, J. R., Crihfield, C. L., & Bwanali, L. (2021) · Analytical and Bioanalytical Chemistry · doi:10.1007/s00216-021-03714-9 · Cited by 6
Qtaish, N. A., Villate-Beitia, I., Gallego, I., Martı́nez, G., Soto‐Sánchez, C., Sainz-Ramos, M., López-Méndez, T. B., Paredes, A. J., Chichón, F. J., Zamarreño, N., et al. (2023) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2023.122968 · Cited by 6
Dashti, R., Yoosefian, M., & Mahani, M. K. (2023) · Journal of Molecular Liquids · doi:10.1016/j.molliq.2023.122993 · Cited by 6
GalNAc- or Mannose-PEG-Functionalized Polyplexes Enable Effective Lectin-Mediated DNA Delivery
Steffens, R. C., Folda, P., Fendler, N. L., Höhn, M., Bücher, K. S., Kempter, S., Snyder, N. L., Hartmann, L., Wagner, E., & Berger, S. (2024) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.3c00546 · Cited by 7
Zeng, C., Liu, Z., Zhang, J., Fang, H., Fang, C., Wang, Y., Seeruttun, S. R., Chen, J., Huang, L., & Wang, W. (2017) · Archives of Medical Science · doi:10.5114/aoms.2017.71064 · Cited by 6
Xu, P., Wang, X., Li, Y., & Qiu, J. (2019) · Viruses · doi:10.3390/v11090820 · Cited by 6
Gene Editing in Human Pluripotent Stem Cells: Recent Advances for Clinical Therapies
Şişli, H. B., Hayal, T. B., Seçkin, S., Şenkal, S., Kıratlı, B., Şahi̇n, F., & Doğan, A. (2019) · Advances in experimental medicine and biology · doi:10.1007/5584_2019_439 · Cited by 6
Godinho, B. M. D. C., & Khvorova, A. (2020) · Portuguese National Funding Agency for Science, Research and Technology (RCAAP Project by FCT) · doi:10.25758/set.2227 · Cited by 6
Technological Approaches to Human Performance Enhancement
Blumenthal, M. S., Hottes, A. K., Foran, C. M., & Lee, M. M. (2021) · RAND Corporation eBooks · doi:10.7249/rra1482-2 · Cited by 6
Incorporating structural changes in mortality improvements for mortality forecasting
Li, J. K. K., & Wong, K. (2020) · Scandinavian Actuarial Journal · doi:10.1080/03461238.2020.1748102 · Cited by 6
Impact of Acetylation, Succinylation, and pH on DNA Packaging in PEI-Based Polyplexes
Uddin, M. N., Dinar, M. A. M., Schrass, L. E., Pack, D. W., & DeRouchey, J. E. (2024) · Biomacromolecules · doi:10.1021/acs.biomac.4c00648 · Cited by 6
Feng, X., & Wang, H. (2025) · ACS Chemical Neuroscience · doi:10.1021/acschemneuro.5c00484 · Cited by 5
Gene therapy: Practical considerations for clinical and operational pharmacy practice
Shay, B., & Storey, M. (2024) · American Journal of Health-System Pharmacy · doi:10.1093/ajhp/zxae036 · Cited by 5
New technologies in gene therapy for inducing immune tolerance in hemophilia A
Borsotti, C., & Follenzi, A. (2018) · Expert Review of Clinical Immunology · doi:10.1080/1744666x.2018.1539667 · Cited by 5
Lima, E. R., Cecchi, C. R., Higuti, E., de Jesus, G. P. P., Gomes, A. M., Zacarias, E. A., Bartolini, P., & Peroni, C. N. (2020) · Molecules · doi:10.3390/molecules25215034 · Cited by 5
Preparing pharmacists to manage gene therapies
Myers, C. J. (2020) · Journal of the American Pharmacists Association · doi:10.1016/j.japh.2020.11.018 · Cited by 5
Cinel, V. D. P., Taketa, T. B., de Carvalho, B. G., de la Torre, L. G., de Mello, L. R., da Silva, E. R., & Han, S. W. (2021) · Biopolymers · doi:10.1002/bip.23432 · Cited by 6
Viral Vectors in Gene Therapy and Clinical Applications
Mahoney, A. L. G., Nassif, N. T., O’Brien, B. A., & Simpson, A. M. (2022) · IntechOpen eBooks · doi:10.5772/intechopen.102559 · Cited by 5
Pérez, D., Moyá, M. L., Bautista, M., León, R., Molina-Márquez, A., Vilà, M., Romero-Azogil, L., Benito, E., García‐Martín, M. D. G., Moreno-Gordillo, P., et al. (2023) · Colloids and Surfaces B Biointerfaces · doi:10.1016/j.colsurfb.2023.113219 · Cited by 5
Takatsuka, S., Kubota, T., Kurashina, Y., Kurihara, S., Hirabayashi, M., Fujioka, M., Okano, H. J., & Onoe, H. (2023) · Biotechnology and Bioengineering · doi:10.1002/bit.28482 · Cited by 5
Dehnavi, S. S., Cembran, A., Mahmoudi, N., Aguilar, L. M. C., Wang, Y., Cheeseman, S., Malagutti, N., Franks, S. J., Long, B. M., Lisowski, L., et al. (2023) · Chemical Engineering Journal · doi:10.1016/j.cej.2023.146857 · Cited by 5
Albuquerque, L. J. C., de Oliveira, F. A., Christoffolete, M. A., Nascimento-Sales, M., Berger, S., Wagner, E., Lächelt, U., & Giacomelli, F. C. (2023) · Journal of Colloid and Interface Science · doi:10.1016/j.jcis.2023.11.003 · Cited by 5
CRISPR-ERA for Switching Off (Onco) Genes
García‐Tuñón, I., Vuelta, E., Pérez‐Ramos, S., Hernández‐Rivas, J. M., Méndez, L., Herrero, M., & Sánchez‐Martín, M. (2019) · IntechOpen eBooks · doi:10.5772/intechopen.80245 · Cited by 5
Jin, W., Badea, I., Leary, S. C., & El‐Aneed, A. (2018) · Journal of Pharmaceutical and Biomedical Analysis · doi:10.1016/j.jpba.2018.10.003 · Cited by 5
Chitosan-Based Ocular Drug Delivery Systems
Natesan, S., Krishnaswami, V., Veedu, S. T., Mohanan, D. P., Kandasamy, R., & Palanichamy, R. (2019) · Springer eBooks · doi:10.1007/978-981-15-0263-7_4 · Cited by 5
Gene therapy in regenerative medicine: latest achievements and actual directions of development
Slobodkina, E. A., Karagyaur, M. N., Balabanyan, V. Y., & Макаревич, П. И. (2020) · Genes and Cells · doi:10.23868/202003001 · Cited by 5
Cell Banking of HEK293T cell line for clinical-grade lentiviral particles manufacturing
Perpiñá, U., Herranz, C., Martín‐Ibáñez, R., Boronat, A., Chiappe, F., Monforte, V., Orpella‐Aceret, G., González, E., Olivé, M., Castellà, M., et al. (2020) · Translational Medicine Communications · doi:10.1186/s41231-020-00075-w · Cited by 5
Nonviral Vehicles for Gene Delivery
Warga, E., Austin-Carter, B., Comolli, N. K., & Elmer, J. J. (2021) · Nano LIFE · doi:10.1142/s1793984421300028 · Cited by 5
Schmeer, M., Schleef, M., Shankar, R., Kobelt, D., & Walther, W. (2022) · Methods in molecular biology · doi:10.1007/978-1-0716-2441-8_17 · Cited by 5
Do, H. D., Marie, C., Bessoles, S., Dhôtel, H., Séguin, J., Larrat, B., Doan, B., Scherman, D., Escriou, V., Hacein‐Bey‐Abina, S., et al. (2022) · Exploration of Medicine · doi:10.37349/etat.2022.00090 · Cited by 5
Recent advances on two-dimensional material-based nanosystems for gene delivery
Wang, M., Li, D., Zhu, J., Liu, J., Yin, Y., Su, Y., Jin, C., Li, J., & Zhang, C. Y. (2024) · APL Materials · doi:10.1063/5.0209799 · Cited by 6
Gene therapy for people with hepatocellular carcinoma
Naing, C. M., Ni, H., Aung, H. H., Htet, N. H., & Nikolova, D. (2024) · Cochrane Database of Systematic Reviews · doi:10.1002/14651858.cd013731.pub2 · Cited by 4
Thiourea-Functional Bioreducible Poly(amido amine)s in Gene Delivery
Elzes, M. R., Si, G., Engbersen, J. F. J., & Paulusse, J. M. J. (2019) · ACS symposium series · doi:10.1021/bk-2019-1309.ch005 · Cited by 5
Akbulut, H., Öcal, M., & Sonugür, F. G. (2015) · InTech eBooks · doi:10.5772/61775 · Cited by 6
Corrigendum: Gene therapy clinical trials worldwide to 2017: An update
(2019) · The Journal of Gene Medicine · doi:10.1002/jgm.3124 · Cited by 5
IRF7 drives macrophages to kill bacteria and improves septic outcomes via autophagy
Chen, G., Li, K., Luo, H., Zhao, L.-X., & Jiang, Y. (2025) · JCI Insight · doi:10.1172/jci.insight.189420 · Cited by 4
Enzyme Replacement Therapy & Other Therapeutic Frontiers in Infantile Metabolic Disorders
Choi, H., Sriram, A., Sargiotto, C., & Fajardo, V. M. (2025) · NeoReviews · doi:10.1542/neo.26-12-081 · Cited by 4
Exosome-Mediated RUNX3 DNA Delivery for Lung Cancer Therapy
Jeon, J., Byun, H., Lee, M. C., Kim, J., Park, S. G., Madrigal‐Salazar, A. D., Gonzalez, M. L., Lopez-Pacheco, M. C., Liu, W., Nasr, M. L., et al. (2025) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.5c15987 · Cited by 4
Engineering immune cells for next-generation therapies
Han, M., Lou, J., Pan, M., Xu, S., Jiang, X., & Jing, W. (2026) · Journal of Controlled Release · doi:10.1016/j.jconrel.2026.115012 · Cited by 4
Recombinant DNA technology and gene therapy
Sivamani, Y., Hegde, S., Bhat, A. R., Sajal, H., & Elayaperumal, S. (2024) · Elsevier eBooks · doi:10.1016/b978-0-443-16013-4.00017-8 · Cited by 4
Recent and Advanced Trends in Cancer Treatments
Chithra, R. A., Elamin, M. O., & Karande, S. (2024) · National Journal of Community Medicine · doi:10.55489/njcm.151220244714 · Cited by 4
Oliynyk, R. T. (2019) · International Journal of Molecular Sciences · doi:10.3390/ijms20133352 · Cited by 3
Surface Modification of Adenovirus Vector to Improve Immunogenicity and Tropism
Gabal, Y., & Ramsey, J. D. (2020) · Methods in molecular biology · doi:10.1007/978-1-0716-0795-4_18 · Cited by 4
Raik, S. V., Mashel, T. V., Muslimov, A. R., Epifanovskaya, O. S., Trofimov, M. A., Poshina, D. N., Lepik, K. V., & Skorik, Y. А. (2021) · Materials · doi:10.3390/ma14092467 · Cited by 4
GMO Medicines and hospital pharmacy practice: a review
Webb, T., & Hong, E. (2021) · Journal of Pharmacy Practice and Research · doi:10.1002/jppr.1742 · Cited by 4
Emerging Gene Therapy Technologies for Retinal Ganglion Cell Neuroprotection
Camacho, D. K., Go, C. C., Chaqour, B., Shindler, K. S., & Ross, A. G. (2023) · Journal of Neuro-Ophthalmology · doi:10.1097/wno.0000000000001955 · Cited by 4
Materials for Gene Delivery Systems
Manohar, S. K., Gowrav, M. P., & Gangadharappa, H. V. (2023) · Springer eBooks · doi:10.1007/978-981-99-2119-5_14 · Cited by 4
Verma, A., & Awasthi, A. (2023) · Pharmaspire · doi:10.56933/pharmaspire.2023.15137 · Cited by 4
Galván‐Salazar, H. R., Delgado‐Machuca, M., Hernández‐Fuentes, G. A., Aurelien-Cabezas, N. S., Rodríguez‐Hernández, A., Veloz, I. G., Mendoza-Hernandez, M. A., Martínez‐Fierro, M. L., Zaizar-Fregoso, S. A., Rodriguez-Sanchez, I. P., et al. (2024) · Microbiology Research · doi:10.3390/microbiolres15030105 · Cited by 4
Metadata
- Authors
- Ginn, S. L., Amaya, A. K., Alexander, I. E., Edelstein, M., & Abedi, M. R.
- Affiliations
- The University of Sydney (AU)
- Chatham House (GB)
- Uppsala University Hospital (SE)
- Corresponding author
- Ginn, Samantha L.
- Venue
- The Journal of Gene Medicine
- Publisher
- Wiley
- Published
- 2018-04-19
- Volume
- 20
- Issue
- 5
- Pages
- e3015-e3015
- ISSN
- 1099-498X (ISSN-L)
- 1521-2254
- OpenAlex
- W2983048398
- Cited by
- 903
- References
- 146
- Citations by year
- 2026: 31 · 2025: 68 · 2024: 86 · 2023: 77 · 2022: 124 · 2021: 141 · 2020: 151 · 2019: 148 · 2018: 36
- FWCI
- 16.66
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- The Journal of Gene Medicine (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Health Sciences › Medicine › Oncology › CAR-T cell therapy research
- Keywords
- gene therapy, clinical trials, clinical trial database, gene therapy vectors, disease indications, chimeric antigen receptor T cells, cancer immunotherapy, genome editing, clinical trial trends
- MeSH terms
- Clinical Trials as Topic, Humans, Neoplasms, Global Health, Genetic Therapy, Immunotherapy, Adoptive, Outcome Assessment, Health Care
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review