Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy

Title:Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy

Volume: 14 Issue: 2

Author(s): Balaji Balakrishnan and Giridhara R. Jayandharan

Affiliation:

Keywords: Adeno-associated virus, gene therapy, integration, serotype, viral vectors.

Abstract: Adeno-associated virus (AAV) based vectors have emerged as important tools for gene therapy in humans. The recent successes seen in Phase I/II clinical trials have also highlighted the issues related to the host and vector-related immune response that preclude the universal application of this promising vector system. A fundamental insight into the biological mechanisms by which AAV infects the host cell and a thorough understanding of the immediate and long-lived cellular responses to AAV infection is likely to offer clues and help design better intervention strategies to improve the therapeutic efficiency of AAV vectors. This article reviews the biology of AAV-host cellular interactions and outlines their application in the development of novel and improved AAV vector systems.

Cite this article as:

Balakrishnan Balaji and Jayandharan R. Giridhara, Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy, Current Gene Therapy 2014; 14 (2) . https://dx.doi.org/10.2174/1566523214666140302193709