AAV

Resolution of hepatic fibrosis after zinc-finger nuclease-mediated gene editing in the PiZ mouse model of human alpha-1-antitrypsin deficiency liver disease
March 7th, 2023
First-in-human in vivo genome editing via AAV-zinc finger nucleases for mucopolysaccharidosis I/II and hemophilia B
November 1st, 2022
Overview of Analytics Needed to Support a Robust Gene Therapy Manufacturing Process
January 19th, 2022
Anion-Exchange HPLC Assay for Separation and Quantification of Empty and Full Capsids in Multiple Adeno-Associated Virus Serotypes
April 23rd, 2021
AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction
August 3rd, 2020