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
Tenchov, R., Bird, R., Curtze, A. E., & Zhou, Q. (2021) · ACS Nano · doi:10.1021/acsnano.1c04996 · Cited by 2,221
Biopharmaceutical benchmarks 2018
Walsh, G. (2018) · Nature Biotechnology · doi:10.1038/nbt.4305 · Cited by 978
Hossain, M. S., Karuniawati, H., Jairoun, A. A., Urbi, Z., Ooi, D. J., John, A., Lim, Y. C., Kibria, K. M. K., Mohiuddin, A. K. M., Ming, L. C., et al. (2022) · Cancers · doi:10.3390/cancers14071732 · Cited by 920
Innovative approaches for cancer treatment: current perspectives and new challenges
Pucci, C., Martinelli, C., & Ciofani, G. (2019) · ecancermedicalscience · doi:10.3332/ecancer.2019.961 · Cited by 864
Mendes, B. B., Conniot, J., Avital, A., Yao, D., Jiang, X., Zhou, X., Sharf-Pauker, N., Xiao, Y., Adir, O., Liang, H., et al. (2022) · Nature Reviews Methods Primers · doi:10.1038/s43586-022-00104-y · Cited by 719
Therapeutic in vivo delivery of gene editing agents
Raguram, A., Banskota, S., & Liu, D. R. (2022) · Cell · doi:10.1016/j.cell.2022.03.045 · Cited by 552
Entering the Modern Era of Gene Therapy
Anguela, X. M., & High, K. A. (2018) · Annual Review of Medicine · doi:10.1146/annurev-med-012017-043332 · Cited by 494
Recent advances in the development of gene delivery systems.
Sung, Y., & Kim, S. (2019) · Biomaterials Research · doi:10.1186/s40824-019-0156-z · Cited by 458
Carriers Break Barriers in Drug Delivery: Endocytosis and Endosomal Escape of Gene Delivery Vectors
Degors, I. M. S., Wang, C., Rehman, Z. U., & Zuhorn, I. S. (2019) · Accounts of Chemical Research · doi:10.1021/acs.accounts.9b00177 · Cited by 436
Goertsen, D., Flytzanis, N. C., Goeden, N., Chuapoco, M. R., Cummins, A., Chen, Y., Fan, Y., Zhang, Q., Sharma, J., Duan, Y., et al. (2022) · Nature Neuroscience · doi:10.1038/s41593-021-00969-4 · Cited by 420
Recent advances in nanomaterial-based synergistic combination cancer immunotherapy
Sang, W., Zhang, Z., Dai, Y., & Chen, X. S. (2019) · Chemical Society Reviews · doi:10.1039/c8cs00896e · Cited by 409
Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality
Hudry, E., & Vandenberghe, L. H. (2019) · Neuron · doi:10.1016/j.neuron.2019.02.017 · Cited by 383
Polymeric Delivery of Therapeutic Nucleic Acids
Kumar, R., Chalarca, C. F. S., Bockman, M. R., Bruggen, C. V., Grimme, C. J., Dalal, R. J., Hanson, M. G., Hexum, J. K., & Reineke, T. M. (2021) · Chemical Reviews · doi:10.1021/acs.chemrev.0c00997 · Cited by 379
Recent Advances in Chitosan-Based Carriers for Gene Delivery
Cao, Y., Tan, Y., Wong, Y. S., Liew, M. W. J., & Venkatraman, S. S. (2019) · Marine Drugs · doi:10.3390/md17060381 · Cited by 380
Polyethylenimine (PEI) in gene therapy: Current status and clinical applications
Casper, J., Schenk, S., Parhizkar, E., Detampel, P., Dehshahri, A., & Huwyler, J. (2023) · Journal of Controlled Release · doi:10.1016/j.jconrel.2023.09.001 · Cited by 360
Gene therapy: Comprehensive overview and therapeutic applications
Sayed, N., Allawadhi, P., Khurana, A., Singh, V., Navik, U., Pasumarthi, S. K., Khurana, I., Banothu, A. K., Weiskirchen, R., & Bharani, K. K. (2022) · Life Sciences · doi:10.1016/j.lfs.2022.120375 · Cited by 365
The approved gene therapy drugs worldwide: from 1998 to 2019
Ma, C.-C., Wang, Z.-L., Xu, T., He, Z.-Y., & Wei, Y.-Q. (2020) · Biotechnology Advances · doi:10.1016/j.biotechadv.2019.107502 · Cited by 310
Therapeutic bacteria to combat cancer; current advances, challenges, and opportunities
Sedighi, M., Bialvaei, A. Z., Hamblin, M. R., Ohadi, E., Asadi, A., Hallajzadeh, M., Lohrasbi, V., Mohammadzadeh, N., Amiriani, T., Krůtová, M., et al. (2019) · Cancer Medicine · doi:10.1002/cam4.2148 · Cited by 312
Moradi, S., Mahdizadeh, H., Šarić, T., Kim, J., Harati, J., Shahsavarani, H., Greber, B., & Moore, J. B. (2019) · Stem Cell Research & Therapy · doi:10.1186/s13287-019-1455-y · Cited by 313
An overview of dynamic covalent bonds in polymer material and their applications
Huang, S., Kong, X., Xiong, Y., Zhang, X., Chen, H., Jiang, W., Niu, Y., Xu, W., & Ren, C. (2020) · European Polymer Journal · doi:10.1016/j.eurpolymj.2020.110094 · Cited by 290
The genome editing revolution: review
Khalil, A. M. (2020) · Journal of Genetic Engineering and Biotechnology · doi:10.1186/s43141-020-00078-y · Cited by 306
In vivo gene delivery mediated by non-viral vectors for cancer therapy
Mohammadinejad, R., Dehshahri, A., Madamsetty, V. S., Zahmatkeshan, M., Tavakol, S., Makvandi, P., Khorsandi, D., Pardakhty, A., Ashrafizadeh, M., Afshar, E. G., et al. (2020) · Journal of Controlled Release · doi:10.1016/j.jconrel.2020.06.038 · Cited by 239
Recent trends in cancer therapy: A review on the current state of gene delivery
Yahya, E. B., & Alqadhi, A. M. (2021) · Life Sciences · doi:10.1016/j.lfs.2021.119087 · Cited by 251
Gene therapy clinical trials, where do we go? An overview
Arabi, F., Mansouri, V., & Ahmadbeigi, N. (2022) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2022.113324 · Cited by 259
Ghosh, S., Brown, A. M., Jenkins, C., & Campbell, K. (2020) · Applied Biosafety · doi:10.1177/1535676019899502 · Cited by 218
Appraisal for the Potential of Viral and Nonviral Vectors in Gene Therapy: A Review
Butt, M. H., Zaman, M., Ahmad, A., Khan, R., Mallhi, T. H., Hasan, M. M., Khan, Y. H., Hafeez, S., Massoud, E. E. S., Rahman, M. H., et al. (2022) · Genes · doi:10.3390/genes13081370 · Cited by 230
Silk Fibroin as a Functional Biomaterial for Drug and Gene Delivery
Tomeh, M. A., Hadianamrei, R., & Zhao, X. (2019) · Pharmaceutics · doi:10.3390/pharmaceutics11100494 · Cited by 215
Cyclodextrin-based delivery systems for chemotherapeutic anticancer drugs: A review
Tian, B., Hua, S., & Liu, J. (2019) · Carbohydrate Polymers · doi:10.1016/j.carbpol.2019.115805 · Cited by 217
Engineered materials for in vivo delivery of genome-editing machinery
Tong, S., Moyo, B., Lee, C. M., Leong, K. W., & Bao, G. (2019) · Nature Reviews Materials · doi:10.1038/s41578-019-0145-9 · Cited by 208
Lipid nanoparticles for siRNA delivery in cancer treatment
Moukhtari, S. H. E., Garbayo, E., Amundarain, A., Pascual‐Gil, S., Carrasco‐Leon, A., Prósper, F., Agirre, X., & Blanco‐Prieto, M. J. (2023) · Journal of Controlled Release · doi:10.1016/j.jconrel.2023.07.054 · Cited by 221
Yang, J., Griffin, A. S., Qiang, Z., & Ren, J. (2022) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-022-01243-0 · Cited by 224
Gene-based therapies for neurodegenerative diseases
Sun, J., & Roy, S. (2021) · Nature Neuroscience · doi:10.1038/s41593-020-00778-1 · Cited by 194
Wang, Y., Xie, Y., Kilchrist, K. V., Li, J., Duvall, C. L., & Oupický, D. (2020) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.9b21214 · Cited by 176
Recent Advances in Lipid Nanoparticles for Delivery of mRNA
Yang, L., Gong, L., Wang, P., Zhao, X., Zhao, F. H., Zhang, Z., Li, Y., & Huang, W. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14122682 · Cited by 190
Towards translational optogenetics
Bansal, A., Shikha, S., & Zhang, Y. (2022) · Nature Biomedical Engineering · doi:10.1038/s41551-021-00829-3 · Cited by 174
Nonviral Gene Delivery with Cationic Glycopolymers
Bruggen, C. V., Hexum, J. K., Tan, Z., Dalal, R. J., & Reineke, T. M. (2019) · Accounts of Chemical Research · doi:10.1021/acs.accounts.8b00665 · Cited by 165
Poddar, A., Conesa, J. J., Liang, K., Dhakal, S., Reineck, P., Bryant, G., Pereiro, E., Riccò, R., Amenitsch, H. W., Doonan, C. J., et al. (2019) · Small · doi:10.1002/smll.201902268 · Cited by 162
Advanced drug delivery 2020 and beyond: Perspectives on the future
Bae, Y. H., & Park, K. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.06.018 · Cited by 160
Natural Polymers Decorated MOF-MXene Nanocarriers for Co-delivery of Doxorubicin/pCRISPR
Rabiee, N., Bagherzadeh, M., Jouyandeh, M., Zarrintaj, P., Saeb, M. R., Mozafari, M., Shokouhimehr, M., & Varma, R. S. (2021) · ACS Applied Bio Materials · doi:10.1021/acsabm.1c00332 · Cited by 157
Recent Advances in Stimulus‐Responsive Nanocarriers for Gene Therapy
Cheng, Y., Li, L., Hu, P., Yang, Y., Wei, W., Deng, X., Wang, L., Tay, F. R., & Ma, J. (2021) · Advanced Science · doi:10.1002/advs.202100540 · Cited by 163
Non-Viral in Vitro Gene Delivery: It is Now Time to Set the Bar!
Bono, N., Ponti, F., Mantovani, D., & Candiani, G. (2020) · Pharmaceutics · doi:10.3390/pharmaceutics12020183 · Cited by 162
Strategies for Targeting Gene Therapy in Cancer Cells With Tumor-Specific Promoters
Montaño-Samaniego, M., Bravo‐Estupiñan, D. M., Méndez-Guerrero, O., Alarcón-Hernández, E., & Ibáñez-Hernández, M. Á. (2020) · Frontiers in Oncology · doi:10.3389/fonc.2020.605380 · Cited by 159
TRIM21—From Intracellular Immunity to Therapy
Foss, S., Bottermann, M., Jonsson, A., Sandlie, I., James, L. C., & Andersen, J. T. (2019) · Frontiers in Immunology · doi:10.3389/fimmu.2019.02049 · Cited by 156
The Current Status of Gene Therapy for the Treatment of Cancer
Belete, T. M. (2021) · Biologics · doi:10.2147/btt.s302095 · Cited by 153
Mohammadinejad, R., Dadashzadeh, A., Moghassemi, S., Ashrafizadeh, M., Dehshahri, A., Pardakhty, A., Sasan, H., Sohrevardi, S. M., & Mandegary, A. (2019) · Journal of Advanced Research · doi:10.1016/j.jare.2019.01.004 · Cited by 145
Novel vectors and approaches for gene therapy in liver diseases
Maestro, S., Weber, N. D., Zabaleta, N., Aldabe, R., & González‐Aseguinolaza, G. (2021) · JHEP Reports · doi:10.1016/j.jhepr.2021.100300 · Cited by 143
Cationic lipids for gene delivery: many players, one goal
Ponti, F., Campolungo, M., Melchiori, C., Bono, N., & Candiani, G. (2021) · Chemistry and Physics of Lipids · doi:10.1016/j.chemphyslip.2020.105032 · Cited by 146
Mitigating off-target effects in CRISPR/Cas9-mediated in vivo gene editing
Han, H. A., Pang, J. K. S., & Soh, B.-S. (2020) · Journal of Molecular Medicine · doi:10.1007/s00109-020-01893-z · Cited by 134
Recent Advances in Preclinical Research Using PAMAM Dendrimers for Cancer Gene Therapy
Tarach, P., & Janaszewska, A. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22062912 · Cited by 141
Cationic Liposomes as Vectors for Nucleic Acid and Hydrophobic Drug Therapeutics
Ewert, K. K., Scodeller, P., Simón‐Gracia, L., Steffes, V. M., Wonder, E. A., Teesalu, T., & Safinya, C. R. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13091365 · Cited by 128
Endocytosis and Organelle Targeting of Nanomedicines in Cancer Therapy
Wang, X., Qiu, Y., Wang, M., Zhang, C.-H., Zhang, T., Zhou, H., Zhao, W., Zhao, W., Xia, G., & Shao, R. (2020) · International Journal of Nanomedicine · doi:10.2147/ijn.s274289 · Cited by 128
Endosomal Escape Pathways for Non-Viral Nucleic Acid Delivery Systems
Liang, W., & Lam, J. K. W. (2012) · InTech eBooks · doi:10.5772/46006 · Cited by 137
Adeno-Associated Viral Vectors in Neuroscience Research
Haggerty, D. L., Grecco, G. G., Reeves, K. C., & Atwood, B. K. (2019) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2019.11.012 · Cited by 135
Chitosan-Based Drug Delivery System: Applications in Fish Biotechnology
Wu, Y., Rashidpour, A., Almajano, M. P., & Metón, I. (2020) · Polymers · doi:10.3390/polym12051177 · Cited by 118
Emerging role of nanoclays in cancer research, diagnosis, and therapy
Peixoto, D., Pereira, I., Pereira‐Silva, M., Veiga, F., Hamblin, M. R., LVOV, Y. M., Liu, M., & Paiva‐Santos, A. C. (2021) · Coordination Chemistry Reviews · doi:10.1016/j.ccr.2021.213956 · Cited by 116
Ahmad, M. Z., Rizwanullah, M., Ahmad, J., Alasmary, M. Y., Akhter, M. H., Abdel‐Wahab, B. A., Warsi, M. H., & Haque, A. (2021) · International Journal of Polymeric Materials · doi:10.1080/00914037.2020.1869737 · Cited by 117
Recent advances in regenerative medicine strategies for cancer treatment
Mansouri, V., Beheshtizadeh, N., Gharibshahian, M., Sabouri, L., Varzandeh, M., & Rezaei, N. (2021) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2021.111875 · Cited by 115
BMP-2 Gene Delivery-Based Bone Regeneration in Dentistry
Park, S., Kim, K.-H., Kim, S., Lee, Y., & Seol, Y. (2019) · Pharmaceutics · doi:10.3390/pharmaceutics11080393 · Cited by 111
Almeida, A. M., Queiroz, J. A., de Sousa, F. P., & Sousa, Â. (2019) · Drug Discovery Today · doi:10.1016/j.drudis.2019.07.011 · Cited by 116
Gene therapy: a double-edged sword with great powers
Tang, R., & Xu, Z. (2020) · Molecular and Cellular Biochemistry · doi:10.1007/s11010-020-03834-3 · Cited by 117
Improved Nucleic Acid Therapy with Advanced Nanoscale Biotechnology
Weng, Y., Huang, Q., Li, C., Yang, Y., Wang, X., Yu, J., Huang, Y., & Liang, X. (2019) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2019.12.004 · Cited by 110
Novel targeting approaches and signaling pathways of colorectal cancer: An insight
Tiwari, A., Saraf, S., Verma, A., Panda, P. K., & Jain, S. (2018) · World Journal of Gastroenterology · doi:10.3748/wjg.v24.i39.4428 · Cited by 109
Perspective on Adenoviruses: Epidemiology, Pathogenicity, and Gene Therapy
Crenshaw, B. J., Jones, L. B., Bell, C. R., Kumar, S., & Matthews, Q. L. (2019) · Biomedicines · doi:10.3390/biomedicines7030061 · Cited by 106
Virus-like nanoparticles as a novel delivery tool in gene therapy
Jeevanandam, J., Pal, K., & Danquah, M. K. (2018) · Biochimie · doi:10.1016/j.biochi.2018.11.001 · Cited by 105
Coatopathies: Genetic Disorders of Protein Coats
Dell’Angelica, E. C., & Bonifacino, J. S. (2019) · Annual Review of Cell and Developmental Biology · doi:10.1146/annurev-cellbio-100818-125234 · Cited by 105
Production of Lentiviral Vectors Using Suspension Cells Grown in Serum-free Media
Bauler, M. N., Roberts, J. K., Wu, C., Fan, B., Ferrara, F., Yip, B. H., Diao, S., Kim, Y., Moore, J., Zhou, S., et al. (2019) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2019.11.011 · Cited by 105
Photoswitchable gRNAs for Spatiotemporally Controlled CRISPR-Cas-Based Genomic Regulation
Moroz, E., Satyapertiwi, D., Ramel, M., Høgset, H., Sunyovszki, I., Liu, Z., Wojciechowski, J. P., Zhang, Y., Grigsby, C. L., Brito, L., et al. (2020) · ACS Central Science · doi:10.1021/acscentsci.9b01093 · Cited by 104
Polymeric Carriers for Nucleic Acid Delivery: Current Designs and Future Directions
Peng, L., & Wagner, E. (2019) · Biomacromolecules · doi:10.1021/acs.biomac.9b00999 · Cited by 102
Impact of predictive, preventive and precision medicine strategies in epilepsy
Nabbout, R., & Kuchenbuch, M. (2020) · Nature Reviews Neurology · doi:10.1038/s41582-020-0409-4 · Cited by 101
Chai, R. J., Werner, H., Li, P. Y., Lee, Y. L., Khaing, N. T., Solovei, I. V., Luu, T. D. A., Sharma, B., Navasankari, R., Maric, M., et al. (2021) · Nature Communications · doi:10.1038/s41467-021-24849-4 · Cited by 98
Recent Advances and Challenges in Gene Delivery Mediated by Polyester-Based Nanoparticles
Piperno, A., Sciortino, M. T., Giusto, E., Montesi, M., Panseri, S., & Scala, A. (2021) · International Journal of Nanomedicine · doi:10.2147/ijn.s321329 · Cited by 97
Grijalvo, S., Puras, G., Sesma, J. Z., Sainz-Ramos, M., Qtaish, N. A., López-Méndez, T. B., Mashal, M., Attia, N., Díaz, D. D., Pons, R., et al. (2019) · Pharmaceutics · doi:10.3390/pharmaceutics11020050 · Cited by 99
Prakoso, D., Lim, S. Y., Erickson, J. R., Wallace, R. S., Lees, J. G., Tate, M., Kiriazis, H., Donner, D. G., Henstridge, D. C., Davey, J. R., et al. (2021) · Cardiovascular Research · doi:10.1093/cvr/cvab043 · Cited by 94
Nanotechnology-Based Strategies to Overcome Current Barriers in Gene Delivery
Mirón-Barroso, S., Domènech, E. B., & Trigueros, S. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22168537 · Cited by 91
Ilovitsh, T., Feng, Y., Foiret, J. L., Kheirolomoom, A., Zhang, H., Ingham, E. S., Ilovitsh, A., Tumbale, S. K., Fite, B. Z., Wu, B., et al. (2020) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1914906117 · Cited by 92
Li, X., Salvia, S. L., Liang, Y., Adamiak, M., Kohlbrenner, E., Jeong, D., Chepurko, E., Ceholski, D. K., López-Gordo, E., Yoon, S., et al. (2023) · Circulation · doi:10.1161/circulationaha.122.063759 · Cited by 92
Gene therapy clinical trials worldwide to 2023—an update
Ginn, S. L., Mandwie, M., Alexander, I. E., Edelstein, M. L., & Abedi, M. R. (2024) · The Journal of Gene Medicine · doi:10.1002/jgm.3721 · Cited by 100
Self-citationThe future of peptides in cancer treatment
Kurrikoff, K., Aphkhazava, D., & Langel, Ü. (2019) · Current Opinion in Pharmacology · doi:10.1016/j.coph.2019.01.008 · Cited by 89
Uchida, S., & Kataoka, K. (2019) · Journal of Biomedical Materials Research Part A · doi:10.1002/jbm.a.36614 · Cited by 90
Peptide-Based Nanoparticles for Therapeutic Nucleic Acid Delivery
Boisguérin, P., Konate, K., Josse, E., Vivès, É., & Deshayes, S. (2021) · Biomedicines · doi:10.3390/biomedicines9050583 · Cited by 91
High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography
Florea, M., Nicolaou, F., Pacouret, S., Zinn, E. M., Sanmiguel, J. C., Andrés‐Mateos, E., Unzu, C., Wagers, A. J., & Vandenberghe, L. H. (2022) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2022.12.009 · Cited by 93
Enhancing the In Vitro and In Vivo Stabilities of Polymeric Nucleic Acid Delivery Nanosystems
Wang, Y., Ye, M., Xie, R., & Gong, S. (2018) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.8b00749 · Cited by 92
Cornetta, K. G., Bonamino, M., Mahlangu, J. N., Mingozzi, F., Rangarajan, S., & Rao, J. G. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.04.002 · Cited by 94
Immunomodulatory effect of mushrooms and their bioactive compounds in cancer: A comprehensive review
Pathak, M. P., Pathak, K., Saikia, R., Gogoi, U., Ahmad, M. Z., Patowary, P., & Das, A. (2022) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2022.112901 · Cited by 91
Dogbey, D. M., Torres, V. E. S., Fajemisin, E. A., Mpondo, L., Ngwenya, T., Akinrinmade, O. A., Perriman, A. W., & Barth, S. (2023) · Drug Delivery and Translational Research · doi:10.1007/s13346-023-01362-3 · Cited by 97
Cationic micelle: A promising nanocarrier for gene delivery with high transfection efficiency
Wang, H., Ding, S., Zhang, Z., Wang, L., & You, Y. (2019) · The Journal of Gene Medicine · doi:10.1002/jgm.3101 · Cited by 85
Developing Protein-Based Nanoparticles as Versatile Delivery Systems for Cancer Therapy and Imaging
Sandra, F. C., Khaliq, N. U., Sunna, A., & Care, A. (2019) · Nanomaterials · doi:10.3390/nano9091329 · Cited by 83
Safe and effective in vivo delivery of DNA and RNA using proteolipid vehicles
Brown, D. W., Wee, P., Bhandari, P., Bukhari, A. B., Grin, L., Vega, H., Hejazi, M., Sosnowski, D. L., Ablack, J., Clancy, E. K., et al. (2024) · Cell · doi:10.1016/j.cell.2024.07.023 · Cited by 84
Optimizing synthetic nucleic acid and protein nanocarriers: The chemical evolution approach
Freitag, F., & Wagner, E. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.03.005 · Cited by 83
Progress and Perspectives in the Development of Lentiviral Vector Producer Cells
Ferreira, M. V., Cabral, E. T., & Coroadinha, A. S. (2020) · Biotechnology Journal · doi:10.1002/biot.202000017 · Cited by 87
Engineering Gene Therapy: Advances and Barriers
Shahryari, A., Burtscher, I., Nazari, Z., & Lickert, H. (2021) · Advanced Therapeutics · doi:10.1002/adtp.202100040 · Cited by 87
Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort
Perocheau, D. P., Cunningham, S. C., Lee, J., Antinao Diaz, J., Waddington, S. N., Gilmour, K., Eaglestone, S., Lisowski, L., Thrasher, A. J., Alexander, I. E., et al. (2019) · Human Gene Therapy · doi:10.1089/hum.2018.098 · Cited by 81
Self-citationDevelopment of nucleic acid vaccines: use of self-amplifying RNA in lipid nanoparticles
Rodríguez‐Gascón, A., del Pozo‐Rodríguez, A., & Solinís, M. Á. (2014) · International Journal of Nanomedicine · doi:10.2147/ijn.s39810 · Cited by 79
Musculoskeletal tissue engineering: Regional gene therapy for bone repair
Collon, K. W., Gallo, M. C., & Lieberman, J. R. (2021) · Biomaterials · doi:10.1016/j.biomaterials.2021.120901 · Cited by 80
Virus-like Nonvirus Cationic Liposome for Efficient Gene Delivery via Endoplasmic Reticulum Pathway
Yuan, X., Qin, B., Yin, H., Shi, Y., Jiang, M., Luo, L., Luo, Z., Zhang, J., Li, X., Zhu, C., et al. (2020) · ACS Central Science · doi:10.1021/acscentsci.9b01052 · Cited by 77
Saiyed, A. N., Vasavada, A. R., & Johar, K. (2022) · Future Journal of Pharmaceutical Sciences · doi:10.1186/s43094-022-00413-9 · Cited by 79
Raftery, R. M., Walsh, D. P., Ferreras, L. A. B., Castaño, I. M., Chen, G., Lemoine, M., Osman, G., Shakesheff, K. M., Dixon, J. E., & O’Brien, F. J. (2019) · Biomaterials · doi:10.1016/j.biomaterials.2019.119277 · Cited by 79
Synthetic multi-layer nanoparticles for CRISPR-Cas9 genome editing
Tang, H., Zhao, X., & Jiang, X. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.03.001 · Cited by 80
Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives
Staal, F. J. T., Aiuti, A., & Cavazzana, M. (2019) · Frontiers in Pediatrics · doi:10.3389/fped.2019.00443 · Cited by 77
Polymer‐mediated gene therapy: Recent advances and merging of delivery techniques
Salameh, J. W., Zhou, L., Ward, S. M., Chalarca, C. F. S., Emrick, T. S., & Figueiredo, M. L. (2019) · Wiley Interdisciplinary Reviews Nanomedicine and Nanobiotechnology · doi:10.1002/wnan.1598 · Cited by 80
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