Phillip W.L. Tai

The paper identifies an amino acid variant in AAV capsid VR-1 that reduces liver targeting, suggesting its potential for minimizing liver toxicity in gene therapy.

AAVone is a single-plasmid system for AAV production that increases yield, reduces DNA impurities, and simplifies the manufacturing process.

This paper establishes standardized nomenclature and reporting for analyzing rAAV vectors using PacBio HiFi sequencing to improve quality assessment and vector design.

This paper develops single-AAV vectors for precise in vivo genome editing using Nme2Cas9 with HDR templates or multiple sgRNAs, showing efficacy in treating HT-I and MPS-I mouse models.