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.
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AAV vectors have an excellent safety record in clinical trials and preclinical animal studies.
“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]
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- 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
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