Area of research
Genetics · Molecular Biology
Research interest
Research interests include Virus-based gene therapy research, CRISPR and Genetic Engineering, CAR-T cell therapy research, and Viral Infectious Diseases and Gene Expression in Insects.
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression
In vivo genome editing of the albumin locus as a platform for protein replacement therapy
Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys
Robust ZFN-mediated genome editing in adult hemophilic mice