Cited by 267Virus-based gene therapy researchRNA Interference and Gene DeliveryViral Infectious Diseases and Gene Expression in Insects

Gene Therapy with Viral Vectors

Kootstra, Neeltje A., Verma, Inder M.·2003·Annual Review of Pharmacology and Toxicology

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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Registry

Crossref · 10.1146/annurev.pharmtox.43.100901.140257

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137 of 137 references verified0 citation errors · 0 not checkable

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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
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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