Key Areas Expertise
AAV Biology and Gene Therapy
Pioneered key discoveries in AAV biology, including the identification of the covalent linkage between the AAV Rep78 protein and the viral genome, a foundational advancement in the field.
Introduced the TNNT2 promoter to the gene therapy field, now widely used for cardiomyocyte-specific gene expression.
Demonstrated that AAV9, delivered early after cardiac myocardial infarction (MI), preferentially transduces cardiomyocytes bordering the infarct zone and attenuates left ventricular remodeling.
Achieved preservation of cardiac function and protection against MI through systemic administration of AAV9 carrying the TNNT2 promoter to drive therapeutic gene expression.
Capsid Engineering
Expertise in rational design and directed evolution to develop tissue-specific AAV capsids, including those targeted to cardiotropic and lung tissues.
Designed specialized AAV capsids for targeted gene delivery to pancreatic tumor cells.
AAV Vector Design
Engineered nephron-segment-specific promoters for precise renal gene expression.
Developed novel delivery methods, such as retrograde ureteral infusion, to enhance targeted AAV delivery.
Mini Gene and Promoter Design
Developed compact promoters and mini-genes to maximise therapeutic payloads in the AAV vectors.
AAV vector production and purification
Experienced in producing and purifying R&D-grade AAV vectors, adhering to high-quality standards for preclinical research.
Study design and gene expression analyses
Experienced in designing in vitro and in vivo pre-clinical studies tailored to project goals.
Performed detailed gene expression analyses to provide meaningful insights.
Oversaw experimental workflows to ensure accurate and reliable outcomes.