Irvin T. Garza

This paper describes a novel AAV9 variant, AAV9fyhr, that improves CNS gene delivery while minimizing peripheral tissue tropism through targeted amino acid modifications.

This paper develops a gene therapy using artificial miRNA delivered via adeno-associated virus to reduce tau in a mouse model of tauopathy, showing efficacy even at late disease stages.

This study shows that AAV-mediated gene therapy in SLC13A5-deficient mice reduces extracellular citrate, normalizes electrophysiologic and sleep issues, and improves seizure resistance.