Area of research
Genetics · Molecular Biology
Research interest
Research interests include Virus-based gene therapy research, RNA Interference and Gene Delivery, Lysosomal Storage Disorders Research, and CRISPR and Genetic Engineering.
Gene Therapy in Patients with the Crigler–Najjar Syndrome
Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler–Najjar Syndrome
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase
Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression
Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics