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

Cancer Treatment Evolution from Traditional Methods to Stem Cells and Gene Therapy

Dong, C., He, W., Li, Q., Lü, Y., Ju, D., Gu, Y., & Zhao, K. (2021) · Current Gene Therapy · doi:10.2174/1566523221666211119110755 · Cited by 40

Histidine‐enhanced gene delivery systems: The state of the art

Hooshmand, S. E., Sabet, M. J., Hasanzadeh, A., Mousavi, S. M. K., Moghaddam, N. H., Hooshmand, S. A., Rabiee, N., Liu, Y., Hamblin, M. R., & Karimi, M. (2022) · The Journal of Gene Medicine · doi:10.1002/jgm.3415 · Cited by 41

Extracellular vesicles over adeno-associated viruses: Advantages and limitations as drug delivery platforms in precision medicine

Yom-Tov, N., Guy, R., & Offen, D. (2022) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2022.114535 · Cited by 42

Calixarene‐Embedded Nanoparticles for Interference‐Free Gene–Drug Combination Cancer Therapy

Liu, Q., Zhang, T., Zheng, Y., Wang, C., Kang, Z., Zhao, Y., Chai, J., Li, H., Guo, D., Liu, Y., et al. (2021) · Small · doi:10.1002/smll.202006223 · Cited by 39

NANOG Decoy Oligodeoxynucleotide–Encapsulated Niosomes Nanocarriers: A Promising Approach to Suppress the Metastatic Properties of U87 Human Glioblastoma Multiforme Cells

Gharbavi, M., Johari, B., Rismani, E., Mousazadeh, N., Taromchi, A. H., & Sharafi, A. (2020) · ACS Chemical Neuroscience · doi:10.1021/acschemneuro.0c00699 · Cited by 38

Bacteriophage-Mediated Cancer Gene Therapy

Petrov, G., Dymova, M., & Richter, V. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms232214245 · Cited by 38

Adenovirus as a Vector and Oncolytic Virus

Matsunaga, W., & Gotoh, A. (2023) · Current Issues in Molecular Biology · doi:10.3390/cimb45060307 · Cited by 43

Translating current biomedical therapies for long duration, deep space missions

Iosim, S., MacKay, M. J., Westover, C. D., & Mason, C. E. (2019) · Precision Clinical Medicine · doi:10.1093/pcmedi/pbz022 · Cited by 39

Adenoviral vector‐based platforms for developing effective vaccines to combat respiratory viral infections

Elkashif, A., Alhashimi, M., Sayedahmed, E. E., Sambhara, S. R., & Mittal, S. K. (2021) · Clinical & Translational Immunology · doi:10.1002/cti2.1345 · Cited by 41

Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges

Zhao, L., Yang, Z., Zheng, M., Shi, L., Gu, M., Liu, G., Miao, F., Chang, Y., Fanghua, H., & Tang, N. (2023) · Genes & Diseases · doi:10.1016/j.gendis.2023.02.010 · Cited by 40

Advanced gene nanocarriers/scaffolds in nonviral-mediated delivery system for tissue regeneration and repair

Zhang, W., Hou, Y., Yin, S., Miao, Q., Lee, K., Zhou, X., & Wang, Y. (2024) · Journal of Nanobiotechnology · doi:10.1186/s12951-024-02580-8 · Cited by 43

Nano-vectors for CRISPR/Cas9-mediated genome editing

Yang, P., Lee, A. Y.-T., Xue, J., Chou, S., Lee, C., Tseng, P., Zhang, T. X., Zhu, Y., Lee, J., Chiou, S., et al. (2022) · Nano Today · doi:10.1016/j.nantod.2022.101482 · Cited by 41

Development of a Spray-Dried Formulation of Peptide-DNA Nanoparticles into a Dry Powder for Pulmonary Delivery Using Factorial Design

Munir, M., Kett, V. L., Dunne, N., & McCarthy, H. O. (2022) · Pharmaceutical Research · doi:10.1007/s11095-022-03256-4 · Cited by 37

Delivery of genome editing tools: A promising strategy for HPV-related cervical malignancy therapy

Aghamiri, S., Talaei, S., Roshanzamiri, S., Zandsalimi, F., Fazeli, E., Aliyu, M., Avarvand, O. K., Ebrahimi, Z., Keshavarz‐Fathi, M., & Ghanbarian, H. (2020) · Expert Opinion on Drug Delivery · doi:10.1080/17425247.2020.1747429 · Cited by 37

Genetically-modified Stem Cell in Regenerative Medicine and Cancer Therapy; A New Era

Verdi, J., Hassanzadeh, A., Shamlou, S., Yousefi, N., & Nikoo, M. (2021) · Current Gene Therapy · doi:10.2174/1566523221666210707125342 · Cited by 36

Influence of charge ratio of liposome/DNA complexes on their size after extrusion and transfection efficiency

Brgles, M., Šantak, M., Halassy, B., Forčić, D., & Tomašić, J. (2012) · International Journal of Nanomedicine · doi:10.2147/ijn.s27471 · Cited by 36

A Polymeric Nanoparticle Formulation for Targeted mRNA Delivery to Fibroblasts

Rodrigues, A. F., Rebelo, C., Simões, S. M. N., Paulo, C., Pinho, S. L. C., Francisco, V., & Ferreira, L. (2022) · Advanced Science · doi:10.1002/advs.202205475 · Cited by 33

Non-viral nucleic acid delivery approach: A boon for state-of-the-art gene delivery

Sahu, K. K., Pradhan, M., Singh, D., Singh, M. R., & Yadav, D. K. (2023) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2023.104152 · Cited by 36

Corneal Regeneration Using Gene Therapy Approaches

Sarkar, S., Panikker, P., D’Souza, S., Shetty, R., Mohan, R. R., & Ghosh, A. (2023) · Cells · doi:10.3390/cells12091280 · Cited by 36

Ligand Decorated Chitosan as an Advanced Nanocarrier for Targeted Delivery: A Critical Review

Qindeel, M., Ahmed, N., Khan, G. M., & Rehman, A. U. (2019) · Nanomedicine · doi:10.2217/nnm-2018-0490 · Cited by 35

Solutions against emerging infectious and noninfectious human diseases through the application of baculovirus technologies

Targovnik, A. M., Simonin, J. A., Callum, G. J. M., Smith, I., Warlet, F. U. C., Nugnes, M. V., Miranda, M. V., & Belaich, M. N. (2021) · Applied Microbiology and Biotechnology · doi:10.1007/s00253-021-11615-1 · Cited by 34

Adenoviral Transduction of Mesenchymal Stem Cells: In Vitro Responses and In Vivo Immune Responses after Cell Transplantation

Treacy, O., Ryan, A. E., Heinzl, T., O’Flynn, L., Cregg, M., Wilk, M. M., Odoardi, F., Lohan, P., O’Brien, T., Nosov, M., et al. (2012) · PLoS ONE · doi:10.1371/journal.pone.0042662 · Cited by 35

Effect of YAP1 silencing on esophageal cancer

Zhao, S., Zhao, J., Li, X., Yang, Y., Zhu, D., Zhang, C., Liu, D., & Wu, K. (2016) · OncoTargets and Therapy · doi:10.2147/ott.s102338 · Cited by 35

Peptide ligands for the affinity purification of adeno‐associated viruses from HEK 293 cell lysates

Chu, W., Shastry, S., Barbieri, E., Prodromou, R., Greback‐Clarke, P., Smith, W. K., Moore, B., Kilgore, R., Cummings, C., Pancorbo, J., et al. (2023) · Biotechnology and Bioengineering · doi:10.1002/bit.28495 · Cited by 33

Stimuli-responsive nanomaterials for the precision delivery of mRNA cancer vaccines

Srivastava, R. (2025) · Nano Trends · doi:10.1016/j.nwnano.2025.100147 · Cited by 36

Gene therapy in epilepsy

Zhang, L., & Wang, Y. (2021) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2021.112075 · Cited by 36

Activation of Innate Immunity by Therapeutic Nucleic Acids

Bishani, A., & Chernolovskaya, E. L. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms222413360 · Cited by 35

Curative Cell and Gene Therapy for Osteogenesis Imperfecta

Schindeler, A., Lee, L. R., O’Donohue, A. K., Ginn, S. L., & Munns, C. F. (2020) · Journal of Bone and Mineral Research · doi:10.1002/jbmr.4549 · Cited by 39

The Magic Staff: A Comprehensive Overview of Baculovirus-Based Technologies Applied to Human and Animal Health

Pidre, M. L., Arrías, P. N., Morales, L. C. A., & Romanowski, V. (2022) · Viruses · doi:10.3390/v15010080 · Cited by 37

A humanized chemogenetic system inhibits murine pain-related behavior and hyperactivity in human sensory neurons

Pérez-Sánchez, J., Middleton, S. J., Pattison, L. A., Hilton, H., Awadelkareem, M. A., Zuberi, S. R., Renke, M. B., Hu, H., Yang, X., Clark, A. J., et al. (2023) · Science Translational Medicine · doi:10.1126/scitranslmed.adh3839 · Cited by 35

Recent advances in aerosol gene delivery systems using non-viral vectors for lung cancer therapy

Lee, A. Y., Cho, M., & Kim, S. (2019) · Expert Opinion on Drug Delivery · doi:10.1080/17425247.2019.1641083 · Cited by 34

Integrated development of enzymatic DNA digestion and membrane chromatography processes for the purification of therapeutic adenoviruses

Kawka, K., Wilton, A. N., Madadkar, P., Medina, M. F. C., Lichty, B. D., Ghosh, R., & Latulippe, D. R. (2020) · Separation and Purification Technology · doi:10.1016/j.seppur.2020.117503 · Cited by 33

Optimizing pDNA Lipo-polyplexes: A Balancing Act between Stability and Cargo Release

Berger, S., Levačić, A. K., Hörterer, E., Wilk, U., Benli‐Hoppe, T., Wang, Y., Öztürk, Ö., Luo, J., & Wagner, E. (2021) · Biomacromolecules · doi:10.1021/acs.biomac.0c01779 · Cited by 33

A Systematic Review and Meta-Analyses of Interventional Clinical Trial Studies for Gene Therapies for the Inherited Retinal Degenerations (IRDs)

Tuohy, G., & Megaw, R. D. (2021) · Biomolecules · doi:10.3390/biom11050760 · Cited by 33

Unveiling the potential of antisense oligonucleotides: Mechanisms, therapies, and safety insights

Ersöz, E., & Demir‐Dora, D. (2024) · Drug Development Research · doi:10.1002/ddr.22187 · Cited by 34

Battling Neurodegenerative Diseases with Adeno-Associated Virus-Based Approaches

Mijanović, O., Branković, A., Borovjagin, A. V., Butnaru, D. V., Bezrukov, E. A., Sukhanov, R. B., Shpichka, A. I., Timashev, P. S., & Ulasov, I. V. (2020) · Viruses · doi:10.3390/v12040460 · Cited by 32

What’s new in IBD therapy: An “omics network” approach

Fiocchi, C., & Iliopoulos, D. (2020) · Pharmacological Research · doi:10.1016/j.phrs.2020.104886 · Cited by 34

NIR light‐triggered nanomaterials‐based prodrug activation towards cancer therapy

Zhang, F., Wu, Q., & Liu, H. (2020) · Wiley Interdisciplinary Reviews Nanomedicine and Nanobiotechnology · doi:10.1002/wnan.1643 · Cited by 32

Global Governance of Human Genome Editing: What Are the Rules?

Marchant, G. E. (2021) · Annual Review of Genomics and Human Genetics · doi:10.1146/annurev-genom-111320-091930 · Cited by 35

The DREADDful Hurdles and Opportunities of the Chronic Chemogenetic Toolbox

Claes, M., Groef, L. D., & Moons, L. (2022) · Cells · doi:10.3390/cells11071110 · Cited by 36

The vicissitudes of gene therapy

Evans, C. H. (2019) · Bone and Joint Research · doi:10.1302/2046-3758.810.bjr-2019-0265 · Cited by 32

Biomimetic gene delivery system coupled with extracellular vesicle–encapsulated AAV for improving diabetic wound through promoting vascularization and remodeling of inflammatory microenvironment

He, S., Li, Z., Xie, L., Lin, R., Yan, B., Li, B., Luo, L., Xv, Y., Wen, H., Liang, Y., et al. (2025) · Journal of Nanobiotechnology · doi:10.1186/s12951-025-03261-w · Cited by 33

The Memory-Modifying Potential of Optogenetics and the Need for Neuroethics

Adamczyk, A. K., & Zawadzki, P. (2020) · NanoEthics · doi:10.1007/s11569-020-00377-1 · Cited by 37

SINEUPs: a novel toolbox for RNA therapeutics

Espinoza, S., Bon, C., Valentini, P., Pierattini, B., Tettey‐Matey, A., Damiani, D., Pulcrano, S., Sanges, R., Persichetti, F., Takahashi, H., et al. (2021) · Essays in Biochemistry · doi:10.1042/ebc20200114 · Cited by 33

DNA nuclear targeting sequences for enhanced non-viral gene transfer: An in vitro and in vivo study

Guen, Y. T. L., Pichon, C., Guégan, P., Pluchon, K., Haute, T., Quemener, S., Ropars, J., Midoux, P., Gall, T. L., & Montier, T. (2021) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2021.03.012 · Cited by 30

Treatment of Melanoma by Nano-conjugate-Delivered Wee1 siRNA

Zhang, X., Cai, A., Gao, Y., Zhang, Y., Duan, X., & Men, K. (2021) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.1c00316 · Cited by 30

A novel ICG-labeled cyclic TMTP1 peptide dimer for sensitive tumor imaging and enhanced photothermal therapy in vivo

Wang, L., Zhang, D., Li, J., Li, F., Wei, R., Jiang, G., Xu, H., Wang, X., Zhou, Y., & Xi, L. (2021) · European Journal of Medicinal Chemistry · doi:10.1016/j.ejmech.2021.113935 · Cited by 30

Smart Strategies for Precise Delivery of CRISPR/Cas9 in Genome Editing

Hasanzadeh, A., Noori, H., Jahandideh, A., Moghaddam, N. H., Mousavi, S. M. K., Nourizadeh, H., Saeedi, S., Karimi, M., & Hamblin, M. R. (2022) · ACS Applied Bio Materials · doi:10.1021/acsabm.1c01112 · Cited by 30

An Engineered Galactosylceramidase Construct Improves AAV Gene Therapy for Krabbe Disease in Twitcher Mice

Pan, X., Sands, S. A., Yue, Y., Zhang, K., LeVine, S. M., & Duan, D. (2019) · Human Gene Therapy · doi:10.1089/hum.2019.008 · Cited by 29

Anti-ageing gene therapy: Not so far away?

Vaiserman, A. M., FALCO, E. D., Koliada, A. K., Maslova, O., & Balistreri, C. R. (2019) · Ageing Research Reviews · doi:10.1016/j.arr.2019.100977 · Cited by 33

Treating Bietti crystalline dystrophy in a high-fat diet-exacerbated murine model using gene therapy

Qu, B., Wu, S., Jiao, G., Zou, X., Li, Z., Guo, L., Sun, X., Huang, C., Sun, Z., Zhang, Y., et al. (2020) · Gene Therapy · doi:10.1038/s41434-020-0159-3 · Cited by 29

The FDA-Approved Drug Nelfinavir Inhibits Lytic Cell-Free but Not Cell-Associated Nonlytic Transmission of Human Adenovirus

Georgi, F., Andriasyan, V., Witte, R., Murer, L., Hemmi, S., Yu, L. X., Grove, M., Meili, N., Kuttler, F., Yakimovich, A., et al. (2020) · Antimicrobial Agents and Chemotherapy · doi:10.1128/aac.01002-20 · Cited by 29

Artificial Intelligence (AI)-Aided Structure Optimization for Enhanced Gene Delivery: The Effect of the Polymer Component Distribution (PCD)

Li, Y., He, Z., Sigen, A., Wang, X., Li, Z., Johnson, M., Foley, R., Lara‐Sáez, I., Lyu, J., & Wang, W. (2023) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.3c05010 · Cited by 31

Negative regulation of cationic nanoparticle-induced inflammatory toxicity through the increased production of prostaglandin E2 via mitochondrial DNA-activated Ly6C+ monocytes

Liu, L., Liu, Y., Xu, B., Liu, C., Jia, Y., Liu, T., Fang, C., Wang, W., Ren, J., He, Z., et al. (2018) · Theranostics · doi:10.7150/thno.21693 · Cited by 30

Optimization of the quality by design approach for gene therapy products: A case study for adeno-associated viral vectors

Tanaka, T., Hanaoka, H., & Sakurai, S. (2020) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2020.08.002 · Cited by 27

The Ocular Gene Delivery Landscape

Sahu, B., Chug, I., & Khanna, H. (2021) · Biomolecules · doi:10.3390/biom11081135 · Cited by 28

The international clinical trials registry platform (ICTRP): data integrity and the trends in clinical trials, diseases, and drugs

Namiot, E. D., Smirnovová, D., Sokolov, A. V., Chubarev, V. N., Tarasov, V. V., & Schiöth, H. B. (2023) · Frontiers in Pharmacology · doi:10.3389/fphar.2023.1228148 · Cited by 32

Lipid-nanoparticle-enabled nucleic acid therapeutics for liver disorders

Arjunan, P., Kathirvelu, D., Mahalingam, G., Goel, A., Zacharaiah, U. G., Srivastava, A., & Marepally, S. K. (2024) · Acta Pharmaceutica Sinica B · doi:10.1016/j.apsb.2024.04.015 · Cited by 33

Exosome-based therapeutics in bone regeneration: from fundamental biology to clinical translation

Tajafrooz, F., Ghofrani, S., Sadeghghomi, F., Chamas, A. E. H., Rahimi, N., Mirakhor, A., Hooshiar, M. H., & Raee, A. (2025) · Stem Cell Research & Therapy · doi:10.1186/s13287-025-04686-8 · Cited by 35

Rational Engineering and Preclinical Evaluation of Neddylation and SUMOylation Site Modified Adeno-Associated Virus Vectors in Murine Models of Hemophilia B and Leber Congenital Amaurosis

Maurya, S., Mary, B., & Jayandharan, G. R. (2019) · Human Gene Therapy · doi:10.1089/hum.2019.164 · Cited by 27

The Protein Corona Does Not Influence Receptor-Mediated Targeting of Virus-like Particles

Suchanova, J. Z., Hejtmánková, A., Neburková, J., Cígler, P., Forstová, J., & Španielová, H. (2020) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.0c00240 · Cited by 28

A comparison of AAV-vector production methods for gene therapy and preclinical assessment

Davidsson, M., Negrini, M., Hauser, S., Svanbergsson, A., Lockowandt, M., Tomasello, G., Manfredsson, F. P., & Heuer, A. (2020) · Scientific Reports · doi:10.1038/s41598-020-78521-w · Cited by 30

Supramolecular Peptide Nanofibrils with Optimized Sequences and Molecular Structures for Efficient Retroviral Transduction

Sieste, S., Mack, T. M., Lump, E., Hayn, M., Schütz, D., Röcker, A., Meier, C. A., Kaygisiz, K., Kirchhoff, F., Knowles, T. P. J., et al. (2021) · Advanced Functional Materials · doi:10.1002/adfm.202009382 · Cited by 27

Gene therapy: challenges in cell culture scale-up

Shupe, J., Zhang, A., Odenwelder, D. C., & Dobrowsky, T. M. (2022) · Current Opinion in Biotechnology · doi:10.1016/j.copbio.2022.102721 · Cited by 28

Clinical holds for cell and gene therapy trials: Risks, impact, and lessons learned

Wills, C. A., Drago, D., & Pietrusko, R. G. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.101125 · Cited by 28

Current Trends in Viral Gene Therapy for Human Orthopaedic Regenerative Medicine

Venkatesan, J. K., Rey‐Rico, A., & Cucchiarini, M. (2019) · Tissue Engineering and Regenerative Medicine · doi:10.1007/s13770-019-00179-x · Cited by 28

Corneal fibrosis: From in vitro models to current and upcoming drug and gene medicines

Cubillo, L. T., Gürdal, M., & Zeugolis, D. I. (2024) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2024.115317 · Cited by 28

Detection of intra‐articular gene therapy in horses using quantitative real time PCR in synovial fluid and plasma

Haughan, J. E., Jiang, Z., Stefanovski, D., Moss, K. L., Ortved, K. F., & Robinson, M. A. (2020) · Drug Testing and Analysis · doi:10.1002/dta.2785 · Cited by 28

BMP gene delivery for skeletal tissue regeneration

Bez, M., Pelled, G., & Gazit, D. (2020) · Bone · doi:10.1016/j.bone.2020.115449 · Cited by 27

Autophagy-inhibiting polymer as an effective nonviral cancer gene therapy vector with inherent apoptosis-sensitizing ability

Wang, J., Zhou, X., Wang, H., Xiao, Q., Ding, K., Dong, X., Xu, S., Shen, B., Sun, J., Zhou, Z., et al. (2020) · Biomaterials · doi:10.1016/j.biomaterials.2020.120156 · Cited by 27

A primer to gene therapy: Progress, prospects, and problems

Zittersteijn, H. A., Gonçalves, M. A. F. V., & Hoeben, R. C. (2020) · Journal of Inherited Metabolic Disease · doi:10.1002/jimd.12270 · Cited by 28

Clinical Applications and Immunological Aspects of Electroporation-Based Therapies

da Luz, J. C. D. S., Antunes, F., Clavijo-Salomon, M. A., Signori, E., Tessarollo, N. G., & Strauss, B. E. (2021) · Vaccines · doi:10.3390/vaccines9070727 · Cited by 28

The efficacy of lapine preconditioned or genetically modified IL4 over-expressing bone marrow-derived mesenchymal stromal cells in corticosteroid-associated osteonecrosis of the femoral head in rabbits

Maruyama, M., Moeinzadeh, S., Guzman, R. A., Zhang, N., Storaci, H. W., Utsunomiya, T., Lui, E., Huang, E. E., Rhee, C., Gao, Q., et al. (2021) · Biomaterials · doi:10.1016/j.biomaterials.2021.120972 · Cited by 27

Dendrimers as Non-Viral Vectors in Gene-Directed Enzyme Prodrug Therapy

Chiş, A. A., Dobrea, C. M., Rus, L. L., Frum, A., Morgovan, C., Butucă, A., Totan, M., Juncan, A. M., Gligor, F. G., & Arseniu, A. M. (2021) · Molecules · doi:10.3390/molecules26195976 · Cited by 32

Novel Developments to Enable Treatment of CNS Diseases with Targeted Drug Delivery

Meyer, A. H., Feldsien, T. M., Mezler, M., Untucht, C., Venugopalan, R., & Lefebvre, D. R. (2023) · Pharmaceutics · doi:10.3390/pharmaceutics15041100 · Cited by 27

A novel plier-like gemini cationic niosome for nucleic acid delivery

Pengnam, S., Patrojanasophon, P., Rojanarata, T., Ngawhirunpat, T., Yingyongnarongkul, B., Radchatawedchakoon, W., & Opanasopit, P. (2019) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2019.04.032 · Cited by 27

Clinical and Translational Landscape of Viral Gene Therapies

Yudaeva, A., Kostyusheva, A., Kachanov, A., Brezgin, S., Ponomareva, N., Parodi, A., Pokrovsky, V. S., Lukashev, A. N., Chulanov, V., & Kostyushev, D. S. (2024) · Cells · doi:10.3390/cells13221916 · Cited by 30

Advancements in Cancer Treatment: Harnessing the Synergistic Potential of Graphene-Based Nanomaterials in Combination Therapy

Keyvani, A. H., Mohammadnejad, P., Pazoki–Toroudi, H., Gilabert, I. P., Chu, T., Manshian, B. B., Soenen, S. J., & Sohrabi, B. (2025) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.4c15536 · Cited by 26

Bioresponsive polyplexes – chemically programmed for nucleic acid delivery

Hager, S., & Wagner, E. (2018) · Expert Opinion on Drug Delivery · doi:10.1080/17425247.2018.1526922 · Cited by 26

Self-assembling Peptides in Current Nanomedicine: Versatile Nanomaterials for Drug Delivery

Peng, F., Zhang, W., & Qiu, F. (2019) · Current Medicinal Chemistry · doi:10.2174/0929867326666190712154021 · Cited by 26

Lipoplex-based therapeutics for effective oligonucleotide delivery: a compendious review

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Liposomal formulations for treating lysosomal storage disorders

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Peptide T7-modified polypeptide with disulfide bonds for targeted delivery of plasmid DNA for gene therapy of prostate cancer

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Gene therapy clinical trials worldwide to 2023—an update

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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