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Annu Rev Pharmacol Toxicol. 2003;43:413-39. Epub 2002 Jan 10.

Gene therapy with viral vectors.

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  • 1Laboratory of Genetics, The Salk Institute, La Jolla, California 92037-1099, USA.


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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