Area of research
Genetics · Physiology
Research interest
Research interests include Virus-based gene therapy research, Lysosomal Storage Disorders Research, RNA Interference and Gene Delivery, and Neurogenetic and Muscular Disorders Research.
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I
Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys
Lessons Learned from the Clinical Development and Market Authorization of Glybera
Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells