Minimum Effective Dose to Achieve Biochemical Correction with Adeno-Associated Virus Vector-Mediated Gene Therapy in Mice with Pompe Disease

Human Gene Therapy, Ahead of Print.

Source: Human Gene Therapy - April 27, 2022 Category: Genetics & Stem Cells Authors: Sang-Oh Han Dorothy Gheorghiu Songtao Li Hye Ri Kang Dwight Koeberl Source Type: research