Hematopoietic stem cell gene therapy for Niemann–Pick disease and other lysosomal storage diseases
✍ Scribed by Edward H Schuchman
- Book ID
- 108313468
- Publisher
- Elsevier Science
- Year
- 1999
- Tongue
- English
- Weight
- 249 KB
- Volume
- 102
- Category
- Article
- ISSN
- 0009-3084
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📜 SIMILAR VOLUMES
## Abstract There are more than 40 different forms of inherited lysosomal storage diseases (LSDs) known to occur in humans and the aggregate incidence has been estimated to approach 1 in 7000 live births. Most LSDs are associated with high morbidity and mortality and represent a significant burden
## Abstract ## Background Glycogen storage disease type II (GSDII) or Pompe disease is an inherited disease of glycogen metabolism caused by a lack of functional lysosomal acid α‐glucosidase (GAA). Affected individuals store glycogen in lysosomes resulting in fatal hypertrophic cardiomyopathy and