Single Vector AAV Approach to Genome Editing in Pompe Disease

TitleSingle Vector AAV Approach to Genome Editing in Pompe Disease
Publication TypeConference Paper
Year of Publication2021
AuthorsB Arnson, J Wang, D Courtney, S-O Han, S Li, BR Cullen, N Bursac, and D Koeberl
Conference NameMolecular Therapy
Date Published04/2021