Gene therapy clinical trials worldwide to 2017: An update

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

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

Polyethylene glycol–modified DOTAP:cholesterol/adenovirus hybrid vectors have improved transduction efficiency and reduced immunogenicity

Vupputuri, S., Tayebi, L., Koralege, R. S. H., Nigatu, A. S., Mozafari, M., Mishra, A., Liu, L., & Ramsey, J. D. (2021) · Journal of Nanoparticle Research · doi:10.1007/s11051-020-05134-9 · Cited by 14

The role of gap junctions in cell death and neuromodulation in the retina

Szarka, G., Balogh, M., Tengölics, Á. J., Ganczer, A., Völgyi, B., & Kovács‐Öller, T. (2021) · Neural Regeneration Research · doi:10.4103/1673-5374.308069 · Cited by 16

A proton/macromolecule-sensing approach distinguishes changes in biological membrane permeability during polymer/lipid-based nucleic acid delivery

Boonstra, E., Hatano, H., Miyahara, Y., Uchida, S., Goda, T., & Cabral, H. (2021) · Journal of Materials Chemistry B · doi:10.1039/d1tb00645b · Cited by 14

Can precision medicine be personal; Can personalized medicine be precise?

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Progress of oncolytic virotherapy for neuroblastoma

Chen, X., Dai, S.-Y., Zhan, Y., Yang, R., Chen, D.-Q., Li, Y., Zhou, E. Y., & Dong, R. (2022) · Frontiers in Pediatrics · doi:10.3389/fped.2022.1055729 · Cited by 14

Gene therapy and nasopharyngeal carcinoma

Hughes, J., Alusi, G., & Wang, Y. (2012) · Rhinology Journal · doi:10.4193/rhino11.239 · Cited by 14

Is Malaysia Ready for Human Gene Editing: A Regulatory, Biosafety and Biosecurity Perspective

Kalidasan, V., & Das, K. T. (2021) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2021.649203 · Cited by 18

Amphiphilic Chitosan Bearing Double Palmitoyl Chains and Quaternary Ammonium Moieties as a Nanocarrier for Plasmid DNA

Pol, T., Chonkaew, W., Hocharoen, L., Niamnont, N., Butkhot, N., Roshorm, Y. M., Kiatkamjornwong, S., Hoven, V. P., & Pratumyot, K. (2022) · ACS Omega · doi:10.1021/acsomega.1c06101 · Cited by 13

Chitosan-Based Nanocomposites for Biological Applications

Yalçın, S., & Çankaya, N. (2022) · IntechOpen eBooks · doi:10.5772/intechopen.106379 · Cited by 13

Hyper-Branched Cationic Cyclodextrin Polymers for Improving Plasmid Transfection in 2D and 3D Spheroid Cells

Monfared, Y. K., Mahmoudian, M., Cecone, C., Caldera, F., Haiaty, S., Heidari, H. R., Rahbarghazi‬, R., Matencio, A., Zakeri‐Milani, P., & Trotta, F. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14122690 · Cited by 13

Engineering aspects of lipid-based delivery systems: In vivo gene delivery, safety criteria, and translation strategies

Eş, İ., Thakur, A., Khaneghah, A. M., Foged, C., & de la Torre, L. G. (2024) · Biotechnology Advances · doi:10.1016/j.biotechadv.2024.108342 · Cited by 13

Novel strategies in HPV‑16‑related cervical cancer treatment: An in vitro study of combined siRNA-E5 with oxaliplatin and ifosfamide chemotherapy

Rasizadeh, R., Aghbash, P. S., Mokhtarzadeh, A., Poortahmasebi, V., Oskouee, M. A., Nahand, J. S., Amini, M., Mahdavi, S. Z. B., Yari, A. H., & Baghi, H. B. (2024) · Gene · doi:10.1016/j.gene.2024.148904 · Cited by 13

Precise Enzymatic Cleavage Sites for Improved Bioactivity of siRNA Lipo-Polyplexes

Reinhard, S., Wang, Y., Dengler, S., & Wagner, E. (2018) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.8b00585 · Cited by 13

Limb-girdle Muscular Dystrophy and Therapy: Insights into Cell and Gene-based Approaches

Taheri, F., Taghizadeh, E., Pour, M. J. R., Rostami, D., Renani, P. G., Rastgar‐Moghadam, A., & Hayat, S. M. G. (2020) · Current Gene Therapy · doi:10.2174/1566523220666200218113526 · Cited by 13

Conditioning Regimens in Long-Term Pre-Clinical Studies to Support Development of Ex Vivo Gene Therapy: Review of Nonproliferative and Proliferative Changes

Chanut, F. J.-A., Sanvito, F., Ferrari, G., Visigalli, I., Carriglio, N., Hernández, R. J., Norata, R., Doglioni, C., Naldini, L., & Cristofori, P. G. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.135 · Cited by 13

Gene Therapy Clinical Trials

Capone, F., Nappi, F., & Galli, M. C. (2020) · Elsevier eBooks · doi:10.1016/b978-0-12-812034-7.00011-x · Cited by 15

ACE2 as therapeutic agent

Li, Q., Grant, M. B., Richards, E. M., & Raizada, M. K. (2020) · Clinical Science · doi:10.1042/cs20200570 · Cited by 13

Gene-Delivery Ability of New Hydrogenated and Partially Fluorinated Gemini bispyridinium Surfactants with Six Methylene Spacers

Massa, M., Rivara, M., Donofrío, G., Cristofolini, L., Peracchia, E., Compari, C., Bacciottini, F., Orsi, D., Franceschi, V., & Fisicaro, E. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms23063062 · Cited by 13

Uso de vetores adenovirais na identificação de grupo de complementação gênica de pacientes com Xeroderma pigmentosum e em animais deficientes em reparo de DNA.

Leite, R. A. (2008) · Digital Library of Theses and Dissertations (Universidade de São Paulo) · doi:10.11606/t.42.2008.tde-27112008-165017 · Cited by 13

Computational genome engineering through AI-CRISPR-precision medicine integration in modern therapeutics

Agboola, O. E., Agboola, S. S., Odeghe, O. B., Olaiya, O. E., Ayinla, Z. A., Akinsanya, P. O., Ilesanmi, O. S., Kehinde, I. O., Adegbuyi, A. T., Kolawole, O. A., et al. (2025) · Annales Pharmaceutiques Françaises · doi:10.1016/j.pharma.2025.08.001 · Cited by 14

Gene Therapy: A New Approach in Modern Medicine

Yazdani, A., Alirezaie, Z., Motamedi, M. J., & Amani, J. (2018) · International Journal of Medical Reviews · doi:10.29252/ijmr-050304 · Cited by 15

A Novel Gene Delivery Vector of Agonistic Anti-Radioprotective 105 Expressed on Cell Membranes Shows Adjuvant Effect for DNA Immunization Against Influenza

Yamazaki, T., Biswas, M., Kosugi, K., Nagashima, M., Inui, M., Tomono, S., Takagi, H., Ichimonji, I., Nagaoka, F., Ainai, A., et al. (2020) · Frontiers in Immunology · doi:10.3389/fimmu.2020.606518 · Cited by 12

Dioscin potentiates the antitumor effect of suicide gene therapy in melanoma by gap junction intercellular communication-mediated antigen cross-presentation

Zhang, W., Lin, L., Zhang, Y., Zhao, T., Zhan, Y., Wang, H., Fang, J., & Du, B. (2022) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2022.112973 · Cited by 12

A Quality by Design Approach in Pharmaceutical Development of Non-Viral Vectors with a Focus on miRNA

Toma, I., Porfire, A. S., Tefas, L. R., Berindan‐Neagoe, I., & Tomuță, I. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14071482 · Cited by 12

Gene therapy approaches for equine osteoarthritis

Thampi, P., Samulski, R. J., Grieger, J. C., Phillips, J. N., McIlwraith, C. W., & Goodrich, L. R. (2022) · Frontiers in Veterinary Science · doi:10.3389/fvets.2022.962898 · Cited by 14

Toward gene therapy for congenital thrombotic thrombocytopenic purpura

Dekimpe, C., Roose, E., Sakai, K., Tersteeg, C., Meyer, S. F. D., & Vanhoorelbeke, K. (2023) · Journal of Thrombosis and Haemostasis · doi:10.1016/j.jtha.2022.12.018 · Cited by 12

Unexplored power of CRISPR-Cas9 in neuroscience, a multi-OMICs review

Banazadeh, M., Abiri, A., Poortaheri, M. M., Asnaashari, L., Langarizadeh, M. A., & Forootanfar, H. (2024) · International Journal of Biological Macromolecules · doi:10.1016/j.ijbiomac.2024.130413 · Cited by 12

The future of complement therapeutics

Kolev, M. V., Rao, K. N., Yeh, M. W., Parikh, A., & Deschatelets, P. (2024) · Exploration of Immunology · doi:10.37349/ei.2024.00161 · Cited by 13

siRNA-based nanotherapeutic approaches for targeted delivery in rheumatoid arthritis

Sawan, S., Kumari, A., Majie, A., Ghosh, A., Karmakar, V., Kumari, N., Ghosh, S., & Gorain, B. (2024) · Biomaterials Advances · doi:10.1016/j.bioadv.2024.214120 · Cited by 15

Genetic advancements in breast cancer treatment: a review

Shokoohi, M., Sedaghatshoar, S., Arian, H., Mokarami, M., Habibi, F., & Bamarinejad, F. (2025) · Discover Oncology · doi:10.1007/s12672-025-01884-x · Cited by 14

Paracrine Interactions Involved in Human Induced Pluripotent Stem Cells Differentiation into Chondrocytes

Zhao, Y., Liu, H., Zhao, C., Dang, P., Li, H., & Farzaneh, M. (2019) · Current Stem Cell Research & Therapy · doi:10.2174/1574888x15666191224122058 · Cited by 12

Non-invasive gene delivery across the blood-brain barrier: present and future perspectives

Kimura, S., & Harashima, H. (2021) · Neural Regeneration Research · doi:10.4103/1673-5374.320981 · Cited by 12

Polymer-based non-viral vectors for gene therapy in the skin

Tortajada, L., Felip‐León, C., & Vicent, M. J. (2022) · Polymer Chemistry · doi:10.1039/d1py01485d · Cited by 12

The diffusion-driven microfluidic process to manufacture lipid-based nanotherapeutics with stealth properties for siRNA delivery

Eş, İ., Malfatti-Gasperini, A. A., & de la Torre, L. G. (2022) · Colloids and Surfaces B Biointerfaces · doi:10.1016/j.colsurfb.2022.112476 · Cited by 14

Synthetic biodegradable polymeric materials in non-viral gene delivery

Panchal, S. S., & Vasava, D. V. (2023) · International Journal of Polymeric Materials · doi:10.1080/00914037.2023.2167081 · Cited by 14

Enhancing pDNA Delivery with Hydroquinine Polymers by Modulating Structure and Composition

Roy, P., Kreofsky, N. W., Brown, M. E., Bruggen, C. V., & Reineke, T. M. (2023) · JACS Au · doi:10.1021/jacsau.3c00126 · Cited by 12

Designing cytochrome P450 enzymes for use in cancer gene therapy

Carrera-Pacheco, S. E., Mueller, A., Puente-Pineda, J. A., Zúñiga-Miranda, J., & Guamán, L. P. (2024) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2024.1405466 · Cited by 12

Purification of therapeutic adenoviruses using laterally-fed membrane chromatography

Kawka, K., Madadkar, P., Umatheva, U., Shoaebargh, S., Medina, M. F. C., Lichty, B. D., Ghosh, R., & Latulippe, D. R. (2019) · Journal of Membrane Science · doi:10.1016/j.memsci.2019.02.056 · Cited by 12

One-pot synthesis of compact DNA silica particles for gene delivery and extraordinary DNA preservation

Ramos‐Valle, A., Marín‐Caba, L., García‐Hevia, L., Correa‐Duarte, M. A., & Fanárraga, M. L. (2023) · Materials Today Advances · doi:10.1016/j.mtadv.2023.100357 · Cited by 11

Cell-cell interaction in the pathogenesis of inherited retinal diseases

Du, X., Butler, A. G., & Chen, H. Y. (2024) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2024.1332944 · Cited by 12

Gene Doping Control Analysis of Human Erythropoietin Transgene in Equine Plasma by PCR-Liquid Chromatography High-Resolution Tandem Mass Spectrometry

Yuen, B. P., Wong, K., So, Y.-M., Kwok, W.-H., Cheung, H. W., Wan, T. S. M., Ho, E. N. M., & Wong, W. (2024) · Analytical Chemistry · doi:10.1021/acs.analchem.4c00247 · Cited by 11

Adenoviral Vectors for Gene Therapy of Hereditary Diseases

Muravyeva, A., & Smirnikhina, S. (2024) · Biology · doi:10.3390/biology13121052 · Cited by 11

Novel Semi-Replicative Retroviral Vector Mediated Double Suicide Gene Transfer Enhances Antitumor Effects in Patient-Derived Glioblastoma Models

Lee, M., Kim, Y.-S., Lee, K., Kang, M., Shin, H., Oh, J.-W., Koo, H., Kim, D., Kim, Y., Kong, D., et al. (2019) · Cancers · doi:10.3390/cancers11081090 · Cited by 11

Prospects of Non-Coding Elements in Genomic DNA Based Gene Therapy

Simna, S. P., & Han, Z. (2021) · Current Gene Therapy · doi:10.2174/1566523221666210419090357 · Cited by 11

Understanding Post Entry Sorting of Adenovirus Capsids; A Chance to Change Vaccine Vector Properties

Daussy, C. F., Pied, N., & Wodrich, H. (2021) · Viruses · doi:10.3390/v13071221 · Cited by 12

Cryo-TEM Reveals the Influence of Multivalent Charge and PEGylation on Shape Transitions in Fluid Lipid Assemblies: From Vesicles to Discs, Rods, and Spheres

Steffes, V. M., Zhang, Z., Ewert, K. K., & Safinya, C. R. (2023) · Langmuir · doi:10.1021/acs.langmuir.3c02664 · Cited by 10

An Overview of the CRISPR-Based Genomic- and Epigenome-Editing System: Function, Applications, and Challenges

Mojarrad, M., Qomi, S. B., & Asghari, A. (2019) · Advanced Biomedical Research · doi:10.4103/abr.abr_41_19 · Cited by 12

Recombinant PAL/PilE/FlaA DNA vaccine provides protective immunity against Legionella pneumophila in BALB/c mice

Chen, Y., Yang, Z., Dong, Y., & Chen, Y. (2020) · BMC Biotechnology · doi:10.1186/s12896-020-00620-3 · Cited by 11

Hepatitis B Virus-Like Particle: Targeted Delivery of Plasmid Expressing Short Hairpin RNA for Silencing the Bcl-2 Gene in Cervical Cancer Cells

Akwiditya, M. A., Yong, C. Y., Yusof, M. T., Mariatulqabtiah, A. R., Ho, K. L., & Tan, W. S. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22052320 · Cited by 11

A Review of Brain-Targeted Nonviral Gene-Based Therapies for the Treatment of Alzheimer’s Disease

Arora, S., Sharma, D., Layek, B., & Singh, J. (2021) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.1c00611 · Cited by 11

Gene Therapy on the Road

Feng, Y. (2019) · Current Gene Therapy · doi:10.2174/1566523219999190426144513 · Cited by 11

Editorial: Non-viral vectors for gene therapy/nucleic acid delivery

Poddar, A., Banerjee‬‬‬‬‬‬‬‬‬, R., & Shukla, R. (2023) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2023.1304769 · Cited by 11

Ultrasound-enhanced gene transfection: vectors, methods, and biomedical applications

Wu, J., Gao, Y., Zhou, H., Yang, Z., Rodriguez, R. D. D., Xu, X., Ma, L., & Qiu, L. (2025) · Ultrasonics Sonochemistry · doi:10.1016/j.ultsonch.2025.107607 · Cited by 11

Fiber manipulation and post-assembly nanobody conjugation for adenoviral vector retargeting through SpyTag-SpyCatcher protein ligation

Kadkhodazadeh, M., Mohajel, N., Behdani, M., Baesi, K., Khodaei, B., Azadmanesh, K., & Arashkia, A. (2022) · Frontiers in Molecular Biosciences · doi:10.3389/fmolb.2022.1039324 · Cited by 10

Perturbation of mRNA splicing in liver cancer: insights, opportunities and challenges

Jia, Q., Sun, X., Li, H., Guo, J., Niu, K., Chan, K. M., Bernards, R., Qin, W., & Jin, H. (2024) · Gut · doi:10.1136/gutjnl-2024-333127 · Cited by 10

Click-Shielded and Targeted Lipopolyplexes

Klein, P. M., & Wagner, E. (2019) · Methods in molecular biology · doi:10.1007/978-1-4939-9670-4_8 · Cited by 10

The advent of automated medicine? The values and meanings of precision

Prainsack, B. (2022) · Oxford University Press eBooks · doi:10.1093/oso/9780198863465.003.0015 · Cited by 11

Advanced Gene Therapy Strategies for the Repair of ACL Injuries

Amini, M., Venkatesan, J. K., Liu, W., Leroux, A., Nguyễn, T. N., Madry, H., Migonney, V., & Cucchiarini, M. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms232214467 · Cited by 12

Exploring nutrient supplementation and bioprocess optimization to improve the production of lentiviral vectors in serum‐free medium suspension cultures

Vaz, T. A. G., Rodrigues, A. F., & Coroadinha, A. S. (2023) · Biotechnology Journal · doi:10.1002/biot.202300212 · Cited by 10

Frequency and Pattern of Worldwide Ocular Gene Therapy Clinical Trials up to 2022

Ameri, H., Kesavamoorthy, N., & Bruce, D. N. (2023) · Biomedicines · doi:10.3390/biomedicines11123124 · Cited by 10

Novel AAV variants with improved tropism for human Schwann cells

Drouyer, M., Chu, T., Labit, E., Haase, F., Navarro, R. G., Nazareth, D., Rosin, N. L., Merjane, J., Scott, S., Cabanes‐Creus, M., et al. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101234 · Cited by 10

Genetic doping: WADA we do about the future of ‘cheating’ in sport?

Brown, J. (2019) · The International Sports Law Journal · doi:10.1007/s40318-019-00141-y · Cited by 10

Peripapillary Intravitreal Injection Improves AAV-Mediated Retinal Transduction

Madrakhimov, S. B., Yang, J. Y., Ahn, D. H., Han, J. W., Ha, T. H., & Park, T. K. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.03.018 · Cited by 10

The cell-penetrating YopM protein-functionalized quantum dot-plasmid DNA conjugate as a novel gene delivery vector

Uğurlu, Ö., Barlas, F. B., Evran, S., & Tımur, S. (2020) · Plasmid · doi:10.1016/j.plasmid.2020.102513 · Cited by 10

Impact of photobiomodulation using four diode laser wavelengths of on cationic liposome gene transfection into pre-osteoblast cells

Hosseinpour, S., Xu, C., & Walsh, L. J. (2021) · Journal of Photochemistry and Photobiology B Biology · doi:10.1016/j.jphotobiol.2020.112108 · Cited by 10

Selecting a Cell Engineering Methodology During Cell Therapy Product Development

Timmins, L. M., Burr, A., Carroll, K., Keefe, R., Teryek, M., Cantolupo, L. J., van der Loo, J. C. M., Heathman, T. R. J., Gormley, A. J., Smith, D. W., et al. (2021) · Cell Transplantation · doi:10.1177/09636897211003022 · Cited by 10

Self-assembled PEGylated amphiphilic polypeptides for gene transfection

Klemm, P., Behnke, M., Solomun, J. I., Bonduelle, C. V., Lecommandoux, S., Traeger, A., & Schubert, S. (2021) · Journal of Materials Chemistry B · doi:10.1039/d1tb01495a · Cited by 10

Novel Fluorinated Spermine and Small Molecule PEI to Deliver Anti-PD-L1 and Anti-VEGF siRNA for Highly Efficient Tumor Therapy

Zhang, Y., Yuan, Z., Jin, Y., Zhang, W., & Yuan, W. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13122058 · Cited by 10

An SI-traceable reference material for virus-like particles

Briones, A., Martos, G., Bedu, M., Choteau, T., Josephs, R. D., Wielgosz, R. I., & Ryadnov, M. G. (2022) · iScience · doi:10.1016/j.isci.2022.104294 · Cited by 10

Multifunctional Delivery Systems for Cancer Gene Therapy

McErlean, E. M., McCrudden, C. M., & McCarthy, H. O. (2015) · InTech eBooks · doi:10.5772/61297 · Cited by 10

Enhancement of adenovirus infection and adenoviral vector-mediated gene delivery by bromodomain inhibitor JQ1

Lv, B., Li, J., Li, M., Zhuo, Y., Ren, K., Li, E., & Yang, G. (2018) · Scientific Reports · doi:10.1038/s41598-018-28421-x · Cited by 10

A Bicistronic Adenoviral Vector Carrying Cytosine Deaminase and Granulocyte-Macrophage Colony-Stimulating Factor Increases the Therapeutic Efficacy of Cancer Gene Therapy

Akbulut, H., Cihan, A. Ç., Şahin, G., Tang, Y., & İçlı, F. (2019) · Human Gene Therapy · doi:10.1089/hum.2018.245 · Cited by 10

Optimized Pre-Clinical Grade Production of Two Novel Lentiviral Vector Pseudotypes for Lung Gene Delivery

Gerpe, M. C. R., van Lieshout, L. P., Domm, J. M., van Vloten, J. P., Datu, J., Ingrao, J. C., Yu, D. L., de Jong, J., Moraes, T. J., Krell, P. J., et al. (2020) · Human Gene Therapy · doi:10.1089/hum.2019.211 · Cited by 10

Impact of gene therapy for canine monogenic diseases on the progress of preclinical studies

Switonski, M. (2020) · Journal of Applied Genetics · doi:10.1007/s13353-020-00554-8 · Cited by 12

Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: A Journey from Mouse Cage to Bed Side

García-Pérez, L., Ordas, A., Canté-Barrett, K., Meij, P., Pike‐Overzet, K., Lankester, A. C., & Staal, F. J. T. (2020) · Pharmaceutics · doi:10.3390/pharmaceutics12060549 · Cited by 11

CRISPR—a challenge for national and international regulation

Nicol, D. (2022) · Oxford University Press eBooks · doi:10.1093/oso/9780198863465.003.0014 · Cited by 9

Tools shaping drug discovery and development

Cheung, E., Xia, Y., Caporini, M. A., & Gilmore, J. L. (2022) · Biophysics Reviews · doi:10.1063/5.0087583 · Cited by 9

Precise Gene Knock‐In Tools with Minimized Risk of DSBs: A Trend for Gene Manipulation

Liu, Y. H., Kong, J., Liu, G., Li, Z., & Xiao, Y. (2024) · Advanced Science · doi:10.1002/advs.202401797 · Cited by 10

Retroviral Vector Technology for Gene Therapy: History, Current Landscape, and Future Prospects

Jaballah, S. A., Ali, L. M., Jehad, M. A., Akhlaq, S., Rizvi, T. A., & Mustafa, F. (2025) · Journal of Molecular Biology · doi:10.1016/j.jmb.2025.169473 · Cited by 10

Vetores virais para uso em terapia gênica

Oliveira, B. D. A., França, E. D. S., Souza, V. G., Vallinoto, A. C. R., & da Silva, A. N. M. R. (2018) · Revista Pan-Amazônica de Saúde · doi:10.5123/s2176-62232018000200008 · Cited by 9

Cell and gene therapy manufacturing capabilities in Australia and New Zealand

O’Sullivan, G. M., Velickovic, Z., Keir, M., Macpherson, J. L., & Rasko, J. E. J. (2019) · Cytotherapy · doi:10.1016/j.jcyt.2019.10.010 · Cited by 9

Combining gene therapy with other therapeutic strategies and imaging agents for cancer theranostics

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Preclinical models for studying corticosteroid‐induced osteonecrosis of the femoral head

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Gene therapy clinical trials worldwide to 2007—an update

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Gene therapy clinical trials worldwide to 2012 – an update

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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

Maurya, P. K., Maurya, N. S., & Mani, A. (2021) · Apple Academic Press eBooks · doi:10.1201/9781003129783-5