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Adeno-associated viral vectors: depend(o)ble stability

Part 1. Genetics

1.7. Gene Therapy

Short Specialist Review

  1. Richard O. Snyder

Published Online: 15 JUL 2005

DOI: 10.1002/047001153X.g107313

Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics

Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics

How to Cite

Snyder, R. O. 2005. Adeno-associated viral vectors: depend(o)ble stability. Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics. 1:1.7:97.

Author Information

  1. University of Florida, Gainesville, FL, USA

Publication History

  1. Published Online: 15 JUL 2005

Abstract

Recombinant adeno-associated viral (rAAV) vectors can mediate the safe and long-term correction of genetic diseases in animal models following a single administration, and have been shown to persist and are safe in humans following delivery to lung, sinus, skeletal muscle, brain, and liver. The physiochemical properties of rAAV vector virions are amenable to industry-accepted manufacturing methodologies, long-term storage, and direct in vivo administration.

Keywords:

  • adeno-associated virus;
  • gene therapy;
  • gene transfer;
  • AAV;
  • transduction;
  • integration