Hematopoietic Stem Cell Gene Therapy
β Scribed by David W. Emery; Tamon Nishino; Ken Murata; Michalis Fragkos; George Stamatoyannopoulos
- Publisher
- Carden Jennings Publishing
- Year
- 2002
- Tongue
- English
- Weight
- 133 KB
- Volume
- 75
- Category
- Article
- ISSN
- 0925-5710
No coin nor oath required. For personal study only.
π SIMILAR VOLUMES
From the time that the genes encoding the defective proteins were cloned for a number of inherited diseases, it became a goal to correct those conditions by restoring the normal gene and thereby, its product. For the inherited disorders affecting the blood and its progenitor cells, the hematopoietic
Numerous incremental technological improvements have occurred recently in the application of therapeutic retrovirus-mediated gene transfer into hematopoietic stem cells (HSCs). Improved transduction efficiencies are now reaching levels that may correct some inherited or acquired disorders. Novel ret
## 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 Ξ±βglucosidase (GAA). Affected individuals store glycogen in lysosomes resulting in fatal hypertrophic cardiomyopathy and