In the mid-1990s, while working as a visiting associate at the National Institutes of Health (NIH), he sequenced AAV3, one of eleven types of AAV vector, and subsequently developed other vectors ...

Tropism Redirection of AAV by Cell-type-specific Expression of RNA (TRACER). This broadly applicable, functional, RNA-based technology allows for rapid in vivo evolution of AAV capsids with ...

Next-generation sequencing allows for critical insights into gene therapy products, which can help streamline and accelerate ...

ddPCR technology then amplifies and detects the ligated oligo pairs, while an ITR-2 assay simultaneously detects the AAV genome. Software analyzes the signals from each capsid type to calculate ...

More information: Maryna V. Ivanchenko et al, PCDH15 Dual-AAV Gene Therapy for Deafness and Blindness in Usher Syndrome Type 1F Models, Journal of Clinical Investigation (2024). DOI: 10.1172/JCI177700 ...