Gene Therapy with Viral Vectors

In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector

Naldini, L., Blömer, U., Gallay, P., Ory, D., Mulligan, R., Gage, F. H., Verma, I. M., & Trono, D. (1996) · Science · doi:10.1126/science.272.5259.263 · Cited by 4,903

Characteristics of a human cell line transformed by DNA from human adenovirus type 5

Graham, F. L., Russell, W. C., Smiley, J., & Nairn, R. (1977) · Journal of General Virology · doi:10.1099/0022-1317-36-1-59 · Cited by 4,793

A Third-Generation Lentivirus Vector with a Conditional Packaging System

Dull, T., Zufferey, R., Kelly, M., Mandel, R. J., Nguyen, M., Trono, D., & Naldini, L. (1998) · Journal of Virology · doi:10.1128/jvi.72.11.8463-8471.1998 · Cited by 3,341

Isolation of a Common Receptor for Coxsackie B Viruses and Adenoviruses 2 and 5

Bergelson, J. M., Cunningham, J. A., Droguett, G., Kurt‐Jones, E. A., Krithivas, A., Hong, J. S., Horwitz, M. S., Crowell, R. L., & Finberg, R. W. (1997) · Science · doi:10.1126/science.275.5304.1320 · Cited by 3,102

Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease

Cavazzana, M., Hacein‐Bey‐Abina, S., Basile, G. D. S., Gross, F., Yvon, E. S., Nusbaum, P., Selz, F., Hue, C., Dogniaux, S., Casanova, J., et al. (2000) · Science · doi:10.1126/science.288.5466.669 · Cited by 2,685

Integrins αvβ3 and αvβ5 promote adenovirus internalization but not virus attachment

Wickham, T. J., Mathias, P., Cheresh, D. A., & Nemerow, G. R. (1993) · Cell · doi:10.1016/0092-8674(93)90231-e · Cited by 2,199

Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo

Zufferey, R., Nagy, D., Mandel, R. J., Naldini, L., & Trono, D. (1997) · Nature Biotechnology · doi:10.1038/nbt0997-871 · Cited by 1,977

Self-Inactivating Lentivirus Vector for Safe and Efficient In Vivo Gene Delivery

Zufferey, R., Dull, T., Mandel, R. J., Bukovsky, A., Quiroz, D., Naldini, L., & Trono, D. (1998) · Journal of Virology · doi:10.1128/jvi.72.12.9873-9880.1998 · Cited by 1,815

Construction of a retrovirus packaging mutant and its use to produce helper-free defective retrovirus

Mann, R., Mulligan, R. C., & Baltimore, D. (1983) · Cell · doi:10.1016/0092-8674(83)90344-6 · Cited by 1,776

T Lymphocyte-Directed Gene Therapy for ADA − SCID: Initial Trial Results After 4 Years

Blaese, R. M., Culver, K. W., Miller, A. D., Carter, C. S., Fleisher, T. A., Clerici, M., Shearer, G. M., Chang, L., Chiang, Y. L., Tolstoshev, P., et al. (1995) · Science · doi:10.1126/science.270.5235.475 · Cited by 1,546

Solid tissues can be manipulated ex vivo and used as vehicles for gene therapy

Hasson, E., Slovatizky, Y., Shimoni, Y., Falk, H., Panet, A., & Mitrani, E. (2005) · The Journal of Gene Medicine · doi:10.1002/jgm.740 · Cited by 30

Chitosan for DNA and gene therapy

Layek, B., & Singh, J. (2016) · Elsevier eBooks · doi:10.1016/b978-0-08-100228-5.00008-0 · Cited by 31

Has Molecular and Cellular Imaging Enhanced Drug Discovery and Drug Development?

Niu, G., & Chen, X. S. (2008) · Drugs in R&D · doi:10.2165/0126839-200809060-00002 · Cited by 30

Specific targeted binding of herpes simplex virus type 1 to hepatocytes via the human hepatitis B virus preS1 peptide

Argnani, R., Boccafogli, L., Marconi, P., & Manservigi, R. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302266 · Cited by 30

Physical Approaches for Nucleic Acid Delivery to Liver

Kamimura, K., & Liu, D. (2008) · The AAPS Journal · doi:10.1208/s12248-008-9067-y · Cited by 29

Design and in vivo characterization of self-inactivating human and non-human lentiviral expression vectors engineered for streptogramin-adjustable transgene expression

Mitta, B. (2004) · Nucleic Acids Research · doi:10.1093/nar/gnh104 · Cited by 29

Hematopoietic stem cell gene therapy with drug resistance genes: an update

Budak-Alpdogan, T., Banerjee, D., & Bertino, J. (2005) · Cancer Gene Therapy · doi:10.1038/sj.cgt.7700866 · Cited by 31

A conditionally replicative adenovirus that codes for a TK-GFP fusion protein (Ad5Δ24TK-GFP) for evaluation of the potency of oncolytic virotherapy combined with molecular chemotherapy

Hakkarainen, T., Hemminki, A., Curiel, D. T., & Wahlfors, J. (2006) · International Journal of Molecular Medicine · doi:10.3892/ijmm.18.4.751 · Cited by 28

Polymers for viral gene delivery

Wang, C., & Pham, P.-T. T. (2008) · Expert Opinion on Drug Delivery · doi:10.1517/17425247.5.4.385 · Cited by 27

Bone Regeneration

Shrivats, A. R., Alvarez, P., Schutte, L., & Hollinger, J. O. (2013) · Elsevier eBooks · doi:10.1016/b978-0-12-398358-9.00055-0 · Cited by 28

Noradrenergic neuron-specific overexpression of nNOS in cardiac sympathetic nerves decreases neurotransmission

Wang, L., Li, D., Plested, C. P., Dawson, T., Teschemacher, A. G., & Paterson, D. J. (2006) · Journal of Molecular and Cellular Cardiology · doi:10.1016/j.yjmcc.2006.05.007 · Cited by 27

Promoter Dependence of Transgene Expression by Lentivirus-Transduced Human Blood–Derived Endothelial Progenitor Cells

Liu, J. W., Pernod, G., Dunoyer-Geindre, S., Fish, R. J., Yang, H., Bounameaux, H., & Kruithof, E. K. O. (2005) · Stem Cells · doi:10.1634/stemcells.2004-0364 · Cited by 27

Phospholipid-Based Artificial Viruses Assembled by Multivalent Cations

Tresset, G., Cheong, W. C. D., Tan, Y. L. S., Boulaire, J., & Lam, Y. M. (2007) · Biophysical Journal · doi:10.1529/biophysj.107.104448 · Cited by 27

Toll-like receptor 2 promiscuity is responsible for the immunostimulatory activity of nucleic acid nanocarriers

Pizzuto, M., Gangloff, M., Scherman, D., Gay, N. J., Escriou, V., Ruysschaert, J., & Lonez, C. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.12.029 · Cited by 26

Polymer‐coated viral vectors: hybrid nanosystems for gene therapy

Rajagopal, P., Duraiswamy, S., Sethuraman, S., Rao, J. G., & Krishnan, U. M. (2018) · The Journal of Gene Medicine · doi:10.1002/jgm.3011 · Cited by 27

Gene Therapy Strategies Towards Immune Tolerance to Treat the Autoimmune Diseases

Siatskas, C., Chan, J. M. K., Field, J., Murphy, K., Nasa, Z., Toh, B., & Alderuccio, F. (2006) · Current Gene Therapy · doi:10.2174/156652306775515600 · Cited by 25

The production of viral vectors designed to express large and difficult to express transgenes within neurons

Holehonnur, R., Lella, S. K., Ho, A., Luong, J. A., & Ploski, J. E. (2015) · Molecular Brain · doi:10.1186/s13041-015-0100-7 · Cited by 27

Hepatitis B virus envelope L protein-derived bio-nanocapsules: Mechanisms of cellular attachment and entry into human hepatic cells

Yamada, M., Oeda, A., Jung, J., Iijima, M., Yoshimoto, N., Niimi, T., Jeong, S., Choi, E. K., Tanizawa, K., & Kuroda, S. (2011) · Journal of Controlled Release · doi:10.1016/j.jconrel.2011.11.004 · Cited by 27

In vivo transduction of HIV-1-derived lentiviral particles engineered for macrolide-adjustable transgene expression

Guarino, B., Weber, C. C., & Fussenegger, M. (2005) · The Journal of Gene Medicine · doi:10.1002/jgm.798 · Cited by 25

Adenoviral Delivery of the EMX2 Gene Suppresses Growth in Human Gastric Cancer

Li, J., Mo, M., Zhao, C. F., Chen, Z., Sheng, Q., Mu, H., Zhang, F., Zhang, Y., Zhi, X., Li, H., et al. (2012) · PLoS ONE · doi:10.1371/journal.pone.0045970 · Cited by 24

Safety and efficacy of adeno‐associated viral vector‐mediated insulin gene transfer via portal vein to the livers of streptozotocin‐induced diabetic Sprague‐Dawley rats

Park, Y. M., Woo, S., Lee, G. T., Ko, J., Lee, Y., Zhao, Z., Kim, H. J., Ahn, C. W., Soo, B., Kim, K., et al. (2005) · The Journal of Gene Medicine · doi:10.1002/jgm.708 · Cited by 25

Generation of Human Induced Pluripotent Stem Cells Bearing an Anti-HIV Transgene by a Lentiviral Vector Carrying an Internal Murine Leukemia Virus Promoter

Kamata, M., Liu, S., Liang, M., Nagaoka, Y., & Chen, I. S. Y. (2010) · Human Gene Therapy · doi:10.1089/hum.2010.050 · Cited by 24

Tunable PEGylation of branch-type PEI/DNA polyplexes with a compromise of low cytotoxicity and high transgene expression: in vitro and in vivo gene delivery

Venault, A., Huang, Y.-C., Lo, J.-C., Chou, C.-J., Chinnathambi, A., Higuchi, A., Chen, W., Chen, W.-Y., & Chang, Y. (2017) · Journal of Materials Chemistry B · doi:10.1039/c7tb01046j · Cited by 24

Lentivirus-AIMP2-DX2 shRNA Suppresses Cell Proliferation by Regulating Akt1 Signaling Pathway in the Lungs of AIMP2 +/– Mice

Hwang, S., Chang, S.-H., Minai‐Tehrani, A., Kim, Y.-S., & Cho, M. (2013) · Journal of Aerosol Medicine and Pulmonary Drug Delivery · doi:10.1089/jamp.2011.0959 · Cited by 23

CpG oligonucleotide as an adjuvant for the treatment of prostate cancer

Lubaroff, D. M., & Karan, D. (2009) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2008.12.005 · Cited by 23

Avian adenovirus vector CELO-TK displays anticancer activity in human cancer cells and suppresses established murine melanoma tumors

Shashkova, E. V., Черенова, Л. В., Kazansky, D. B., & Doronin, K. (2005) · Cancer Gene Therapy · doi:10.1038/sj.cgt.7700822 · Cited by 23

In situ stem cell therapy: novel targets, familiar challenges

Agrawal, S., & Schaffer, D. V. (2004) · Trends in biotechnology · doi:10.1016/j.tibtech.2004.12.004 · Cited by 22

Induction of Broadly Neutralising HCV Antibodies in Mice by Integration-Deficient Lentiviral Vector-Based Pseudotyped Particles

Deng, Y., Guan, J., Wen, B., Zhu, N., Chen, H., Song, J., Yang, Y., Wang, Y., & Tan, W. (2013) · PLoS ONE · doi:10.1371/journal.pone.0062684 · Cited by 21

Delivery of transcription factors as modulators of cell differentiation

Rilo-Alvarez, H., Ledo, A. M., Vidal, A., & García-Fuentes, M. (2021) · Drug Delivery and Translational Research · doi:10.1007/s13346-021-00931-8 · Cited by 22

Evaluation of biolistic gene transfer methods in vivo using non-invasive bioluminescent imaging techniques

Xia, J., Martinez, A., Daniell, H., & Ebert, S. N. (2011) · BMC Biotechnology · doi:10.1186/1472-6750-11-62 · Cited by 20

Non-integrating lentiviral vectors based on the minimal S/MAR sequence retain transgene expression in dividing cells

Xu, Z., Chen, F., Zhang, L., Lu, J., Xu, P., Liu, G., Xie, X., Mu, W., Wang, Y., & Liu, D. (2016) · Science China Life Sciences · doi:10.1007/s11427-016-0067-0 · Cited by 20

Selected Strategies for the Delivery of siRNA In Vitro and In Vivo

Laufer, S. D., Detzer, A., Sczakiel, G., & Restle, T. (2010) · RNA technologies · doi:10.1007/978-3-642-12168-5_2 · Cited by 21

Local gene transfer to calcified tissue cells using prolonged infusion of a lentiviral vector

Wazen, R. M., Moffatt, P., Zalzal, S., Daniel, N. G., Westerman, K. A., & Nanci, A. (2006) · Gene Therapy · doi:10.1038/sj.gt.3302824 · Cited by 20

2-diethylaminoethyl-dextran methyl methacrylate copolymer nonviral vector: still a long way toward the safety of aerosol gene therapy

Zarogoulidis, P., Hohenforst‐Schmidt, W., Darwiche, K., Krauss, L., Sparopoulou, D., Sakkas, L., Gschwendtner, A., Huang, H., Turner, F. J., Freitag, L., et al. (2013) · Gene Therapy · doi:10.1038/gt.2013.27 · Cited by 19

Cyclic nucleotide regulation of cardiac sympatho‐vagal responsiveness

Li, D., & Paterson, D. J. (2016) · The Journal of Physiology · doi:10.1113/jp271827 · Cited by 18

Clinical Protocol: Phase I Study of an Adenovirus/Prostate-Specific Antigen Vaccine in Men with Metastatic Prostate Cancer

Lubaroff, D. M., Konety, B. R., Link, B. K., Ratliff, T. L., Madsen, T., Shannon, M., Ecklund, D. J., & Williams, R. D. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.220 · Cited by 18

Dynamic lineage analysis of embryonic morphogenesis using transgenic quail and 4D multispectral imaging

Bower, D. V., Sato, Y., & Lansford, R. D. (2011) · genesis · doi:10.1002/dvg.20754 · Cited by 17

Advances in viral vector-based delivery systems for gene therapy: a comprehensive review

Singh, K., Jain, D., Sethi, P., Gupta, J. K., Dubey, A., Noman, A. A., lal, S., Sharma, P., & Abdallah, E. M. (2025) · 3 Biotech · doi:10.1007/s13205-025-04366-7 · Cited by 21

Radioprotective gene therapy

Maier, P., Veldwijk, M. R., & Wenz, F. K. (2011) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.2011.580271 · Cited by 18

Lentivirus-Based Genetic Manipulations in Neurons In Vivo

Dittgen, T., Licznerski, P., & Osten, P. (2006) · Frontiers in neuroscience · doi:10.1201/9780203486283.ch13 · Cited by 17

Combining stem cells and exon skipping strategy to treat muscular dystrophy

Meregalli, M., Farini, A., & Torrente, Y. (2008) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.8.8.1051 · Cited by 17

Effective transduction of primary mouse blood- and bone marrow-derived monocytes/macrophages by HIV-based defective lentiviral vectors

Zeng, L., Yang, S., Wu, C., Ye, L., & Lu, Y. (2006) · Journal of Virological Methods · doi:10.1016/j.jviromet.2005.12.006 · Cited by 17

Linear Cationic Click Polymers/DNA Nanoparticles: In Vitro Structure–Activity Relationship and In Vivo Evaluation for Gene Delivery

Gao, Y., Yin, Q., Chen, L., Zhang, Z., & Li, Y. (2011) · Bioconjugate Chemistry · doi:10.1021/bc200008j · Cited by 16

Stimuli-responsive polymers in gene delivery

Pişkın, E. (2005) · Expert Review of Medical Devices · doi:10.1586/17434440.2.4.501 · Cited by 16

REVIEW: Synergies of microtissue design, viral transduction and adjustable transgene expression for regenerative medicine

Kelm, J. M., Kramer, B. P., Gonzalez‐Nicolini, V., Ley, B., & Fussenegger, M. (2004) · Biotechnology and Applied Biochemistry · doi:10.1042/ba20030124 · Cited by 16

Long‐term effect of neuronal nitric oxide synthase over‐expression on cardiac neurotransmission mediated by a lentiviral vector

Wang, L., Li, D., Dawson, T., & Paterson, D. J. (2009) · The Journal of Physiology · doi:10.1113/jphysiol.2009.172866 · Cited by 15

A TLR4 agonist synergizes with dendritic cell-directed lentiviral vectors for inducing antigen-specific immune responses

Liang, X., Kim, J. T., Lim, M., Dai, B., Yang, L., Reed, S. G., Baltimore, D., & Wang, P. (2012) · Vaccine · doi:10.1016/j.vaccine.2012.01.074 · Cited by 17

Pseudotyping Lentiviral Vectors with Aura Virus Envelope Glycoproteins for DC-SIGN–Mediated Transduction of Dendritic Cells

Froelich, S., Tai, A., Kennedy, K. L., Zubair, A., & Wang, P. (2011) · Human Gene Therapy · doi:10.1089/hum.2010.196 · Cited by 14

Ultrasound-mediated gene transfection in vitro: Effect of ultrasonic parameters on efficiency and cell viability

Zhang, Y., Tachibana, R., Okamoto, A., Azuma, T., Sasaki, A., Yoshinaka, K., Tei, Y., Takagi, S., & Matsumoto, Y. (2012) · International Journal of Hyperthermia · doi:10.3109/02656736.2012.665568 · Cited by 14

Selective eradication of cancer cells by delivery of adenovirus-based toxins

Shapira, S., Shapira, A., Kazanov, D., Hevroni, G., Kraus, S. R., & Arber, N. (2017) · Oncotarget · doi:10.18632/oncotarget.16934 · Cited by 14

Generation of lentiviral transgenic rats expressing Glutamate Receptor Interacting Protein 1 (GRIP1) in brain, spinal cord and testis

Nakagawa, T., Feliú-Mójer, M. I., Wulf, P. S., Lois, C., Sheng, M., & Hoogenraad, C. C. (2005) · Journal of Neuroscience Methods · doi:10.1016/j.jneumeth.2005.08.001 · Cited by 14

Enhancement of chitosan-mediated gene delivery through combination with phiC31 integrase

Oliveira, A. V., Silva, G. A., & Chung, D. C. (2015) · Acta Biomaterialia · doi:10.1016/j.actbio.2015.01.013 · Cited by 13

Safeguards for Using Viral Vector Systems in Human Gene Therapy: A Resource for Biosafety Professionals Mitigating Risks in Health Care Settings

Brown, A. M., Blind, J. E., Campbell, K., & Ghosh, S. (2020) · Applied Biosafety · doi:10.1177/1535676020934917 · Cited by 13

Bioreducible polyspermine as less toxic and efficient gene carrier

Park, H., Nichols, J. W., Kang, H. C., & Bae, Y. H. (2014) · Polymers for Advanced Technologies · doi:10.1002/pat.3269 · Cited by 12

Double UP: A Dual Color, Internally Controlled Platform for in utero Knockdown or Overexpression

Taylor, R. J., Carrington, J., Gerlach, L. R., Taylor, K. L., Richters, K. E., & Dent, E. W. (2020) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2020.00082 · Cited by 12

Thérapie génique du déficit immunitaire combiné sévère lié à l’X

Fischer, A., Hacein‐Bey‐Abina, S., & Cavazzana, M. (2004) · médecine/sciences · doi:10.1051/medsci/2004201115 · Cited by 12

Applications of tissue-specific and cancer-selective gene promoters for cancer diagnosis and therapy

Kumar, A., Das, S. K., Emdad, L., & Fisher, P. B. (2023) · Advances in cancer research · doi:10.1016/bs.acr.2023.03.005 · Cited by 11

Influence of Immune Responses in Gene/Stem Cell Therapies for Muscular Dystrophies

Farini, A., Sitzia, C., Erratico, S., Meregalli, M., & Torrente, Y. (2014) · BioMed Research International · doi:10.1155/2014/818107 · Cited by 12

Pseudotyping Lentiviral Vectors with Lymphocytic Choriomeningitis Virus Glycoproteins for Transduction of Dendritic Cells and In Vivo Immunization

Zhang, C., Hu, B., Liang, X., Liu, Y., & Wang, P. (2014) · Human Gene Therapy Methods · doi:10.1089/hgtb.2014.105 · Cited by 11

Nanoparticle-mediated delivery of non-viral gene editing technology to the brain

Williams, L., & Larsen, J. (2023) · Progress in Neurobiology · doi:10.1016/j.pneurobio.2023.102547 · Cited by 10

Cell-type specific regulation of cortical excitability through the allatostatin receptor system

Ikrar, T., Shi, Y., Velasquez, T., Goulding, M., & Xu, X. (2012) · Frontiers in Neural Circuits · doi:10.3389/fncir.2012.00002 · Cited by 10

Polyfection as Nonviral Gene Transfer Method —Design of Novel Nonviral Vector Using α-Cyclodextrin—

Arima, H. (2004) · YAKUGAKU ZASSHI · doi:10.1248/yakushi.124.451 · Cited by 10

Adeno-associated viral serotypes differentially transduce inhibitory neurons within the rat amygdala

de Solis, C. A., Hosek, M. P., Holehonnur, R., Ho, A., Banerjee, A., Luong, J. A., Jones, L. E., Chaturvedi, D., & Ploski, J. E. (2017) · Brain Research · doi:10.1016/j.brainres.2017.07.023 · Cited by 9

High efficiency gene delivery into laryngeal muscle with bidirectional electroporation

Saito, K., Lehar, M., Li, Z. B., Braga, N., KING, A. D., Samlan, R. A., & Flint, P. W. (2006) · Otolaryngology · doi:10.1016/j.otohns.2006.04.003 · Cited by 9

Prolongation of heart allograft survival after long-term expression of soluble MHC class I antigens and vIL-10 in the liver by AAV-plasmid-mediated gene transfer

Doenecke, A., Frank, E., Scherer, M. N., Schlitt, H. J., & Geissler, E. K. (2008) · Langenbeck s Archives of Surgery · doi:10.1007/s00423-008-0298-2 · Cited by 9

Delivering miRNA modulators for cancer treatment

Estanqueiro, M., Vasconcelos, M. H., Lobo, J. M. S., & Amaral, M. H. (2018) · Elsevier eBooks · doi:10.1016/b978-0-12-813689-8.00014-8 · Cited by 9

Engineering Baculo- and Lentiviral Vectors for Enhanced and Targeted Gene Delivery

Kaikkonen, M. U. (2008) · Cited by 8

Current advances in combining stem cell and gene therapy for neurodegenerative diseases

Hwang, J.-Y., Won, J.-S., Nam, H., Lee, H. W., & Joo, K. M. (2018) · Precision and Future Medicine · doi:10.23838/pfm.2018.00037 · Cited by 7

In Vivo Expression of Reprogramming Factor OCT4 Ameliorates Myelination Deficits and Induces Striatal Neuroprotection in Huntington’s Disease

Yu, J.-H., Nam, B.-G., Kim, M., Pyo, S., Seo, J. H., & Cho, S. (2021) · Genes · doi:10.3390/genes12050712 · Cited by 9

The potential impact of gene therapy on health-related quality of life (HRQoL) domains in haemophilia

Bullinger, M., Latibeaudiere, D., Lewis, H. B., Miesbach, W., Nolte, S., O’Hara, J., O’Mahony, B., Pollard, D., Skinner, M. W., & Quinn, J. (2021) · The Journal of Haemophilia Practice · doi:10.17225/jhp00176 · Cited by 7

Avasimibe can cooperate with a DC-targeting and integration-deficient lentivector to induce stronger HBV specific T cytotoxic response by regulating cholesterol metabolism

Ma, S., Lv, M., Chen, X., Zang, G., Tang, Z., Zhang, Y., & Hu, W. (2023) · Antiviral Research · doi:10.1016/j.antiviral.2023.105662 · Cited by 7

Glutamate Transporter Activators as Anti-Nociceptive Agents

Stephens, R. L. (2011) · Eurasian Journal of Medicine · doi:10.5152/eajm.2011.39 · Cited by 7

Gene Therapy for Rett Syndrome: Prospects and Challenges

Gadalla, K. K. E., Ross, P. D., Hector, R. D., Bahey, N. G., Bailey, M. E. S., & Cobb, S. (2015) · Future Neurology · doi:10.2217/fnl.15.29 · Cited by 7

Current Concepts in Gene Therapy of the Musculoskeletal System

Ivković, A., Pascher, A., Hudetz, D., Jelić, M., Hašpl, M., Windhager, R. H., & Pecina, M. M. (2006) · Acta chirurgiae orthopaedicae et traumatologiae Cechoslovaca · doi:10.55095/achot2006/016 · Cited by 7

Advances in microbial decorations and its applications in drug delivery

He, T., Zhao, Z., Luo, Z., Jia, W. P., Zhang, J., Zhao, Y., Xiao, W., Ming, Z., & Chen, K. (2023) · Acta Materia Medica · doi:10.15212/amm-2023-0036 · Cited by 7

A Tetracycline-regulated Cell Line Produces High-titer Lentiviral Vectors that Specifically Target Dendritic Cells

Bryson, P. D., Zhang, C., Lee, C.-L., & Wang, P. (2013) · Journal of Visualized Experiments · doi:10.3791/50606 · Cited by 6

A NANOSCALE POLYNUCLEOTIDE-NEUTRAL LIPOSOME SELF-ASSEMBLIES FORMULATED FOR THERAPEUTIC GENE DELIVERY

Científico, C., Süleymanoğlu, E., Biomed, R. E., Biomed, E. J., Moro, E., & Múñiz, P. (2004) · Cited by 6

Construction of traceable cucurbit[7]uril-based virus-mimicking quaternary complexes with aggregation-induced emission for efficient gene transfection

Liu, D., Yan, X., An, J., Ma, J., & Gao, H. (2020) · Journal of Materials Chemistry B · doi:10.1039/d0tb01180k · Cited by 6

Breakthroughs in cancer gene therapy

Liu, K. (2003) · Seminars in Oncology Nursing · doi:10.1016/s0749-2081(03)00049-4 · Cited by 6

A single thiourea group is not enough to get stable thiourea lipoplexes

Kabha, E., Jacquement, C., Pembouong, G., Mignet, N., Scherman, D., & Herscovici, J. (2008) · Bioorganic & Medicinal Chemistry · doi:10.1016/j.bmc.2008.01.029 · Cited by 6

Genetic modification of T cells for cancer therapy.

Imai, C., & Campana, D. (2004) · PubMed · Cited by 6

Transgenic Mice in Biomedical Research

Voncken, J. W., & Hofker, M. H. (2006) · Wiley-VCH eBooks · doi:10.1002/3527600906.mcb.200400155 · Cited by 6

Ultrasound-Mediated Gene Transfection In vitro: Enhanced Efficiency by Complexation of Plasmid DNA

Zhang, Y., Tachibana, R., Okamoto, A., Azuma, T., Sasaki, A., Yoshinaka, K., Osada, K., Kataoka, K., Takagi, S., & Matsumoto, Y. (2012) · Japanese Journal of Applied Physics · doi:10.1143/jjap.51.07gf29 · Cited by 5

Lentiviral Vectors for Gene Delivery to the Nervous System

Eleftheriadou, I., & Mazarakis, N. D. (2015) · Neuromethods · doi:10.1007/978-1-4939-2306-9_2 · Cited by 5

Preparation and phase behaviour of surface-active pharmaceuticals: self-assembly of DNA and surfactants with membranes. Differential adiabatic scanning microcalorimetric study

Süleymanoğlu, E. (2005) · Il Farmaco · doi:10.1016/j.farmac.2005.05.010 · Cited by 5

Diminished Memory T-Cell Expansion Due to Delayed Kinetics of Antigen Expression by Lentivectors

Furmanov, K., Elnekave, M., Sa'eed, A., Segev, H., Eli‐Berchoer, L., Kotton, D. N., Bachrach, G., & Hovav, A. (2013) · PLoS ONE · doi:10.1371/journal.pone.0066488 · Cited by 5

Pharmacokinetic Study of Viral Vectors for Gene Therapy: Progress and Challenges

Xu, X., Jingwen, Y., & Yuan-guo, C. (2011) · InTech eBooks · doi:10.5772/21259 · Cited by 6

Porphyrin-based MOFs for Gene Delivery in Cancer Therapy: Recent Advances and Progress

Kabiri, S., Rahimi, R., Mozafari, M. R., & Naghib, S. M. (2025) · Current Pharmaceutical Design · doi:10.2174/0113816128359818250407020852 · Cited by 4

Rett syndrome: advances in Understanding MeCP2 function, potential gene therapies, and public health implications

Ali, N. E., Tariq, N., Naz, G., Abalkhail, A., Kausar, T., Mazhar, I., Zia, S. K., Aqib, A. I., & Khan, N. U. (2025) · Molecular Biology Reports · doi:10.1007/s11033-025-10802-x · Cited by 5

Multiple platforms of a HIV-2 derived lentiviral vector for expanded utility

Chande, A. G., Raina, S., Dhamne, H., Kamat, R. H., & Mukhopadhyaya, R. (2012) · Plasmid · doi:10.1016/j.plasmid.2012.09.006 · Cited by 4

Lentivirally overexpressed T-bet regulates T-helper cell lineage commitment in chronic hepatitis B patients

Liu, X., Tang, Z., Zhang, Y., Hu, J., Li, D., Zang, G., & Yu, Y. (2012) · Molecular Medicine Reports · doi:10.3892/mmr.2012.905 · Cited by 4

Novel Disulfide-Containing Poly(ß-amino ester)-Functionalised Magnetic Nanoparticles for Efficient Gene Delivery

Liu, Y., Li, S., Feng, L., Yu, H., Qi, X., Wei, W., Li, J., & Dong, W. (2015) · Australian Journal of Chemistry · doi:10.1071/ch15293 · Cited by 4

Bioactive Inorganic and Organic Composite Materials for Bone Regeneration and Gene Delivery

Zhang, Y., & Wu, C. (2013) · doi:10.1201/b13926-12 · Cited by 4

Gene-Silencing for Treatment of Cardiovascular Diseases

Nolte, A., Schneider, M., Walker, T. L., & P., H. (2011) · InTech eBooks · doi:10.5772/10659 · Cited by 4

Lung Gene Therapy: Clinical and Regulatory Issues

Amrite, A. C., Cheruvu, N. P. S., Sundaram, S., Aravalli, R. N., Cheng, P., & Kompella, U. B. (2004) · Clinical Research and Regulatory Affairs · doi:10.1081/crp-120039559 · Cited by 4

Vector Systems for Prenatal Gene Therapy: Choosing Vectors for Different Applications

Coutelle, C., & Waddington, S. N. (2012) · Methods in molecular biology · doi:10.1007/978-1-61779-873-3_3 · Cited by 4

Transfer of Drug Resistance Genes into Hematopoietic Stem Cells for Marrow Protection

Bertino, J. R. (2008) · The Oncologist · doi:10.1634/theoncologist.2008-0173 · Cited by 4

Mechanisms and applications of stem cell gene therapy in autoimmunity

Chan, J. M. K., Siatskas, C., Field, J., Toh, B., & Alderuccio, F. (2006) · Drug Discovery Today Disease Mechanisms · doi:10.1016/j.ddmec.2006.05.003 · Cited by 4

Understanding the prospective of gene therapy for the treatment of breast cancer

Ritika, R., Rani, S., Malviya, R., Rajput, S., Sridhar, S. B., & Kaushik, D. (2025) · Revista de Senología y Patología Mamaria · doi:10.1016/j.senol.2025.100683 · Cited by 4

Baculovirus dual-phase infection strategy and biotechnological applications: A structural and regulatory review

Yuan, X., Wang, C., He, Y., & Fu, Y. (2025) · Biotechnology Advances · doi:10.1016/j.biotechadv.2025.108627 · Cited by 3

First published online as a Review in Advance on March 11, 2005 GENE THERAPY: Twenty-First Century Medicine

Verma, I. M., & Weitzman, M. D. (2005)

GENE THERAPY: Twenty-First Century Medicine

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

A Review on Gene Therapy

Pravalika, G., Maheshwari, K., Divya, B., Shirisha, G., & Balakoti, J. (2024) · International Journal of Pharmaceutical and Biological Science Archive

Gene Therapy: The Molecular Bandage for Treating Genetic Disorders

Karim, M. M. (1970) · Anwer Khan Modern Medical College Journal · doi:10.3329/akmmcj.v3i1.10110

Viral mediated gene therapy

Murphy, B. J. (1999) · Springer eBooks · doi:10.1007/978-94-017-0926-2_19 · Cited by 2