AAV Vector Design and Gene Therapy
Education and Training:
Vanderbilt University, 2006
University of North Carolina, 2006-2008 (under Jude Samulski)Special Interests:
Central Nervous System gene therapy, specifically for Giant Axonal Neuropathy, Rett Syndrome, and Lysosomal Storage Diseases
My core expertise is in AAV gene therapy vector engineering, followed by optimizing approaches to deliver a gene to the central and peripheral nervous system. As reagents have been developed to achieve global and efficient CNS gene delivery, my research focus has also included preclinical studies to apply these reagents toward the development of treatments for neurological diseases. Currently these include Rett Syndrome, Giant Axonal Neuropathy, Tay-Sachs, Krabbe, AGU, and Batten Disease. My future directions include 1) continued development and optimization of AAV vectors specifically tailored toward CNS disorders, 2) testing novel gene therapy approaches for applicable CNS disorders, and 3) facilitating the translation of these approaches from bench to clinic.