Liver

ZFN-Mediated In Vivo Gene Editing in Hepatocytes Leads to Supraphysiologic α-Gal A Activity and Effective Substrate Reduction in Fabry Mice
March 26th, 2021
AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction
August 3rd, 2020