The DOI matches its Crossref record; 137 of the 138 references it cites were found in a registry or an index.
Gene Therapy with Viral Vectors
Abstract
A key factor in the success of gene therapy is the development of gene delivery systems that are capable of efficient gene transfer in a broad variety of tissues, without causing any pathogenic effect. Currently, viral vectors based on many different viruses have been developed, and their performance and pathogenicity has been evaluated in animal models. The results of these studies form the basis for the first clinical trials for correcting genetic disorders using retroviral, adenoviral, and adeno-associated viral vectors. Even though the results of these trials are encouraging, vector development is still required to improve and refine future treatment of hereditary disorders.
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
“AAV vectors have an excellent safety record in clinical trials and preclinical animal studies.”
From Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges (2020) · cites this paper as [2]
Citations
Cites
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
Self-citationCharacteristics 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
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
Research works printed but not linked: 7
- 10.1073/pnas.050581197 · DOI · Paper page
- Kearns WG, 1996, Gene Ther., 3, 748
- Shenk TE. 2001. Adenoviridae: the viruses and their replication. See Ref.140, pp. 2265–300
- Miller CR, 1998, Cancer Res., 58, 5738
- Roizman B, Knipe DM. 2001. Herpes simplex viruses and their replication. See Ref.140, pp. 2399–460
- Hoogerbrugge PM, 1996, Gene Ther., 3, 179
- Knipe DM, Howley PM, eds. 2001. Fields' Virology, Vol. 2. Philadelphia: Lippincott, Williams & Wilkins
Cited by
Sorted by citations
Items 201–267 of 267
Gene Therapy and Virus-Based Cancer Vaccines
Salem, M. L., Gadalla, K. K. E., Fielding, B. C., & Thorne, S. H. (2014) · Springer eBooks · doi:10.1007/978-3-662-44946-2_7 · Cited by 3
Than, P. A., Davis, C. R., Rennert, R. C., Morrison, S. D., Findlay, M. W., Kay, M. A., & Gurtner, G. C. (2021) · Plastic & Reconstructive Surgery · doi:10.1097/prs.0000000000008630 · Cited by 3
Coexpression of TP53, BIM, and PTEN Enhances the Therapeutic Efficacy of Non-Small-Cell Lung Cancer
Lu, L., Ma, D., & Xi, Z. (2024) · Biomacromolecules · doi:10.1021/acs.biomac.3c00988 · Cited by 3
Plasma-mediated transfection of RPE
Palanker, D., Chalberg, T. W., Vankov, A., Huie, P., Molnar, F. E., Butterwick, A., Calos, M. P., Marmor, M. F., & Blumenkranz, M. S. (2006) · Proceedings of SPIE, the International Society for Optical Engineering/Proceedings of SPIE · doi:10.1117/12.649624 · Cited by 3
Problems, Side Effects, and Disappointments in Clinical Cancer Gene Therapy
Liu, T., & Kirn, D. H. (2007) · Humana Press eBooks · doi:10.1007/978-1-59745-222-9_20 · Cited by 3
Shenoi, R. A. (2018) · Elsevier eBooks · doi:10.1016/b978-0-08-101750-0.00010-6 · Cited by 3
Bryson, P. D., & Wang, P. (2013) · Elsevier eBooks · doi:10.1016/b978-0-12-394295-1.00024-x · Cited by 3
Ultrasound-Mediated Gene TransfectionIn 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:10.7567/jjap.51.07gf29 · Cited by 3
RNA-based gene therapies for myotonic dystrophy type 1
Langlois, M. (2003) · Cited by 3
Subcloning of NGF Gene into pSecTag2/Hygro Secretory Vector and Expression in PC12 Cell Line
Kondori, B. J., Asadi, M. H., & Azemati, F. (2015) · The Neuroscience Journal of Shefaye Khatam · doi:10.18869/acadpub.shefa.3.1.21 · Cited by 2
Ligand-directed tumor targeting with hybrid viral phage nanoparticles
Dalal, B., Quinn, T. J., Foster, L., Lin, M. I., Matthews, M., & Yuhan, B. T. (2018) · Elsevier eBooks · doi:10.1016/b978-0-12-813689-8.00013-6 · Cited by 2
Genetics of Hypertension and Cardiovascular Disease
Katsuya, T., Harrap, S. B., & Ogihara, T. (2010) · International Journal of Hypertension · doi:10.4061/2010/951254 · Cited by 2
Applications of Small-Animal Molecular Imaging in Drug Development
Niu, G., & Chen, X. S. (2014) · Springer eBooks · doi:10.1007/978-1-4939-0894-3_24 · Cited by 2
Sehgal, L., Budnar, S., Bhatt, K., Sansare, S., Mukhopadhaya, A., Kalraiya, R. D., & Dalal, S. N. (2012) · PubMed · Cited by 2
Combining Stem Cells and Exon Skipping Strategy to Treat Muscular Dystrophy
Meregalli, M., Farini, A., & Torrente, Y. (2009) · Springer eBooks · doi:10.1007/978-1-4419-1207-7_15 · Cited by 2
Current Trends in the Development of Wound Dressings, Biomaterials and Devices
Banerjee, A., & Bhattacharyya, J. (2021) · Elsevier eBooks · doi:10.1016/b978-0-12-820352-1.00117-6 · Cited by 2
Xi, Y., Jiang, Q., Dai, W. H., Chen, C., Wang, Y., Miao, X., Lai, K., Jiang, Z., Yang, G., & Wang, Y. (2025) · Journal of Zhejiang University SCIENCE B · doi:10.1631/jzus.b2300531 · Cited by 1
RETRACTED: Comprehensive Review on Role of Nanoparticles in Gene Therapy
Vediya, P., Zalavadiya, I., Trivedi, U., Mehta, D. M., & Reddy, B. R. G. (2025) · E3S Web of Conferences · doi:10.1051/e3sconf/202561904009 · Cited by 1
RetractedEx Vivo Gene Therapy for Spinal Fusion
Kaito, T., Stephanie, S. C., & Choi, J. H.-K. (2011) · InTech eBooks · doi:10.5772/18764 · Cited by 1
Seidlits, S. K., Hlavaty, K. A., & Shea, L. D. (2014) · Cambridge University Press eBooks · doi:10.1017/cbo9780511997839.029 · Cited by 1
Bioactive Inorganic and Organic Composite Materials for Bone Regeneration and Gene Delivery
Zhang, Y., & Wu, C. (2013) · CRC Press eBooks · doi:10.1201/b13926-8 · Cited by 1
Hao, X. (2006) · Figshare · Cited by 1
The Blood – Brain Barrier and its Effect on Absorption and Distribution
de Boer, A. G., & Gaillard, P. J. (2010) · Wiley eBooks · doi:10.1002/9780470571224.pse041 · Cited by 1
Thérapie génique du déficit immunitaire combiné sévère lié à LX
Fischer, A. (1997) · Hematology and Cell Therapy · doi:10.1007/s00282-997-0097-1 · Cited by 1
Süleymanoğlu, E. (2006) · Iranian journal of pharmaceutical research · doi:10.22037/ijpr.2010.648 · Cited by 1
Engineering Viruses for CNS studies
Lansford, R. D. (2009) · Elsevier eBooks · doi:10.1016/b978-008045046-9.02006-4 · Cited by 1
New Technologies in Cardiovascular Research: Gene Therapy
Hedman, M., Turunen, M. P., & Ylä‐Herttuala, S. (2006) · Springer eBooks · doi:10.1007/0-387-23329-6_5 · Cited by 1
Chromosomal integration in vitro and in vivo of high capacity adenoviral vectors
Stephen, S. L. (2007) · Kölner Universitäts PublikationsServer (Universität zu Köln)
Cell-Delivered Gene Therapy for HIV
Ledger, S., Savkovic, B., Millington, M., Impey, H. L., Boyd, M. P., John, M., & Symonds, G. P. (2011) · InTech eBooks · doi:10.5772/22658
IMMOBILIZED COBALT AFFINITY PURIFICATION FOR HSV-1 BASED GENE THERAPY VECTORS
Jiang, C. (2006) · D-Scholarship@Pitt (University of Pittsburgh)
Progress toward a combined bacterial and viral gene delivery system for mammalian cells
Simper, M. (2004) · Texas ScholarWorks (Texas Digital Library)
Ligand-Directed AAVP to Deliver a Therapeutic Transgene to Neuroendocrine Tumors of the Pancreas
Smith, T. L. (2013) · Digital Commons-TMC (Texas Medical Center)
Zeng, L., Ye, L., & Lu, Y. (2007) · Virologica Sinica · doi:10.1007/s12250-007-0002-7
Biotechnological Applications of Artificial Microtissues
Sánchez-Bustamante, C. D. (2009) · TDX (Tesis Doctorals en Xarxa)
Vascular Endothelial Growth Factors B and C: Gene Regulation and Gene Transfer In Vivo
Enholm, B. (2004) · Työväentutkimus Vuosikirja
Controlling Nonviral Gene Delivery through the Cell-Biomaterial Interface
Duensing, B. A. (2010) · Lincoln (University of Nebraska)
Structure and Assembly of Membrane-Containing dsDNA Bacteriophages
Huiskonen, J. T. (2005) · Työväentutkimus Vuosikirja
Ivković, A., Pascher, A., Hudetz, D., Jelić, M., Hašpl, M., Windhager, R. H., & Pecina, M. M. (2006)
Doenecke, A., Frank, E., Scherer, M. N., Schlitt, H. J., & Geissler, E. K. (2008) · Langenbecks Archiv für Chirurgie. Supplement · doi:10.1007/978-3-540-78833-1_73
HIV-derived lentiviral vectors engineered for regulated transgene expression
Guarino, B. M. (2005) · Repository for Publications and Research Data (ETH Zurich) · doi:10.3929/ethz-a-005063290
Iannucci, V. (2013) · Ghent University Academic Bibliography (Ghent University)
Linear and Branched Chitosan Oligomers as Delivery Systems for pDNA and siRNA In Vitro and In Vivo
Issa, M. (2006) · KTH Publication Database DiVA (KTH Royal Institute of Technology)
Gene Therapy Applications for Parkinson’s Disease
Torres, E. M., Monville, C., & Dunnett, S. B. (2006) · CRC Press eBooks · doi:10.3109/9781420016390-10
Bacteriophage-Targeted Nanomedicine and Molecular Imaging
Wang, T., & Narayanaswamy, R. (2014) · Frontiers in nanobiomedical research · doi:10.1142/9789814520652_0060
Retrovirus-mediated gene therapy for Farber disease.
Ramsubir, S. (2008) · TSpace (University of Toronto)
ŠTARGARDTO GELTONOSIOS DĖMĖS DISTROFIJOS SKIRTINGOS FENOTIPINĖS IŠRAIŠKOS
Liutkevičienė, R., Glebauskienė, B., Ašmonienė, V., Gelžinis, A., Kriaučiūnienė, L., Vaičiulienė, R., Trumpaitis, J., & Žemaitienė, R. (2016) · Medicinos teorija ir praktika · doi:10.15591/mtp.2016.007
Doenecke, A., Frank, E., Scherer, M. N., & Geissler, E. K. (2008)
Cellular Therapies for Wiskott-Aldrich Syndrome
Braun, C. J., & Klein, C. (2016) · Springer eBooks · doi:10.1007/978-3-319-39562-3_18
Xia, J. (2011) · Journal of International Crisis and Risk Communication Research
Efficient Template-Based Nanomanufacturing of Carbon Nanotube Arrays for Cell Applications
Ahmed, A. (2017) · RIT Scholar Works (Rochester Institute of Technology)
Gene Therapy and Free Flaps for Therapeutic Reconstructive Surgery
Davis, C. R., Than, P. A., & Gurtner, G. C. (2018) · Springer eBooks · doi:10.1007/978-3-319-78957-6_7
Double UP: efficient in utero electroporation via internal controlsPreprint
Taylor, R. J., Carrington, J., Taylor, K. L., Richters, K. E., Gerlach, L. R., & Dent, E. W. (2019) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/571323
An improved lentivirus vector for safer transgene integration and protein transduction
Schenkwein, D. (2013) · UEF eRepo (University of Eastern Finland)
TREATMENT POSSIBILITIES FOR ACQUIRED AND HEREDITARY DISEASES BY GENE THERAPY: A REVIEW
KUMARI, P. V. K., & EKSHITHA, V. H. G. (2021) · International Journal of Applied Pharmaceutics · doi:10.22159/ijap.2021v13i2.39995
Lentiviral Vector Mediated Gene Transfer as an Antiviral and Antitumoral Intervention Strategy
Raina, S. (2015) · Shodhganga
Min, W. (2004) · Journal of Molecular and Cellular Cardiology · doi:10.1016/j.yjmcc.2003.12.003
Khan, M. M. (2016) · Springer eBooks · doi:10.1007/978-3-319-30273-7_11
Khan, M. M. (2008) · Springer eBooks · doi:10.1007/978-0-387-77976-8_10
Lubaroff, D. M., Konety, B. R., Link, B. K., Link, T. L., Madsen, T., Shannon, M., Ecklund, D. J., & Williams, R. D. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.ft-168
Widespread Transduction of Mouse Neocortical Neurons by Subarachnoid Injection of AAV2
Smirnova, M. P., Osipova, A. A., Borodinova, A. A., Bagrinovtseva, T. M., Dobryakova, Y. V., Смирнов, И. В., & Malyshev, A. Y. (2025) · Journal of Visualized Experiments · doi:10.3791/68235
Polymeric Delivery of DNA‐Encoded Biologics
Ting, J., Dykeman-Bermingham, P. A., Cairns, C. E., Petersen, S. R., Snell, N. J., Miskin, R. P., Fisher, J. D., Murthy, S. K., Kevlahan, S., Guzman, G., et al. (2026) · Macromolecular Chemistry and Physics · doi:10.1002/macp.202500421
Stargardt's Macular Dystrophy different phenotipical aspects
Liutkevičienė, R., Glebauskienė, B., Ašmonienė, V., Gelžinis, A., Kriaučiūnienė, L., Vaičiulienė, R., Trumpaitis, J., & Žemaitienė, R. (2015) · Lithuanian University of Health Sciences
Lentivirus and shRNA models for the delivery and regulation of bone sialoprotein functional domains
Johar, D., & Abdulrahman, M. S. (2026) · Next research. · doi:10.1016/j.nexres.2026.102037
Viral vectors in drug delivery: Design and applications
Chouhan, N. K., Sharma, M., Rawat, M., Talati, M. N., Kaur, H., Sharma, A., & Vinchurkar, K. (2026) · Elsevier eBooks · doi:10.1016/b978-0-443-40620-1.00009-1
Design of artificial microtissues
Kelm, J. M. (2005) · Repository for Publications and Research Data (ETH Zurich) · doi:10.3929/ethz-a-005064702
Metadata
- Authors
- Kootstra, N. A., & Verma, I. M.
- Affiliations
- Salk Institute for Biological Studies (US)
- Venue
- Annual Review of Pharmacology and Toxicology
- Publisher
- Annual Reviews
- Published
- 2003-04
- Volume
- 43
- Issue
- 1
- Pages
- 413-439
- ISSN
- 0362-1642 (ISSN-L)
- 1545-4304
- OpenAlex
- W2156754275
- Cited by
- 267
- References
- 138
- Citations by year
- 2026: 3 · 2025: 8 · 2024: 1 · 2023: 5 · 2022: 1 · 2021: 6 · 2020: 6 · 2019: 1 · 2018: 9 · 2017: 4 · 2016: 8 · 2015: 12 · 2014: 15 · 2013: 15 · 2012: 14
- FWCI
- 2.78
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- The Annual Review of Pharmacology and Toxicology (publishedVersion)
- PubMed (publishedVersion)
- Data Archiving and Networked Services (DANS) (submittedVersion)
- Pure Amsterdam UMC (submittedVersion)
- Funders
- American Cancer Society
- Wayne and Gladys Valley Foundation
- Fordham University
- 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
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Viral Infectious Diseases and Gene Expression in Insects
- Keywords
- gene therapy, viral vectors, gene delivery systems, retroviral vectors, adenoviral vector, adeno-associated virus vectors, clinical trials, genetic disorders
- MeSH terms
- Genetic Vectors, Humans, Viruses, Genetic Therapy, Gene Transfer Techniques
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