Title: | A Novel Gene Therapy Approach for GSD III Using an AAV Vector Encoding a Bacterial Glycogen Debranching Enzyme |
Journal : | Molecular therapy. Methods & clinical development, 18 |
Authors: | Lim JA ; Choi SJ ; Gao F ; Kishnani PS ; Sun B |
Material Type: | Article |
Publication Date: | 2020 |
Size: | p 240 |
Languages: | English |
Keywords: | AAV;pullulanase;gene therapy;glycogen debranching enzyme;glycogen storage disease type III; |
Pubmed / DOI : | Pubmed : 32637453 / DOI : 10.1016/j.omtm.2020.05.034 |
N° Profil MNM : | 2020071 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/32637453 |