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.