John A. Chiorini, Ph.D., Chief
NATIONAL INSTITUTES OF HEALTH/NIDCR
10 Center Drive
Building 10 Room 1N113
Bethesda, MD 20892-1190
Phone: (301) 496-4279
Fax: (301) 402-1228
E-mail: jchiorini@mamil.nih.gov
Mission
The overall research goal of the AAV Biology Unit is to define the interactions of adeno-associated virus (AAV) with its target cell. Our hypothesis is that by understanding these interactions, and the biology of the virus, we can contribute to the use of AAV vectors for gene therapy. The Molecular Physiology and Therapeutics Branch¹s program on gene transfer to the salivary gland considers AAV vectors to be the most useful vector for clinical studies with this tissue. Staff members focus on two types of interactions: those encompassing viral transduction of the target cell, and those involving the Rep proteins of wild type AAV and their cellular partners. Current projects study the tropism and transduction pathways of different serotypes of AAV, the lifecycle of the virus, identifying and characterizing new isolates of AAV, and characterizing the interactions between novel cellular proteins and the AAV Rep proteins. Application of this technology include deliver of therapeutic genes to the salivary glands, lung, CNS, inner ear, and eye for the benefit of both local and systemic diseases.