|
|||||
|
|
Antibody formation and mannose‐6‐phosphate receptor expression impact the efficacy of muscle‐specific transgene expression in murine Pompe disease |
|
| Authors: | Baodong Sun Songtao Li Andrew Bird Haiqing Yi Alex Kemper Beth L. Thurberg Dwight D. Koeberl |
| Affiliation: | 1. Division of Medical Genetics, Duke University Medical Centre, Durham, NC, USA;2. Department of Pediatrics/Program on Pediatric Health Services Research, Duke University Medical Centre, Durham, NC, USA;3. Genzyme Corporation, Framingham, MA, USA |
| Abstract: | |
| Keywords: | acid α ‐glucosidase acid maltase adeno‐associated virus antibody formation gene therapy glycogen storage disease type II Pompe disease |