Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy
Balaji Balakrishnan and Giridhara R. Jayandharan
Affiliation: Department of Hematology & Centre for Stem Cell Research, Christian Medical College, Vellore-632004, Tamil Nadu, India.
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.
Keywords: Adeno-associated virus, gene therapy, integration, serotype, viral vectors.
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