Back to Search
Start Over
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy
- Source :
- Journal of Clinical Investigation. August 1, 2013, Vol. 123 Issue 8, p3254, 18 p.
- Publication Year :
- 2013
-
Abstract
- For most lysosomal storage diseases (LSDs) affecting the CNS, there is currently no cure. The BBB, which limits the bioavailability of drugs administered systemically, and the short half-life of lysosomal [...]
Details
- Language :
- English
- ISSN :
- 00219738
- Volume :
- 123
- Issue :
- 8
- Database :
- Gale General OneFile
- Journal :
- Journal of Clinical Investigation
- Publication Type :
- Academic Journal
- Accession number :
- edsgcl.341937521
- Full Text :
- https://doi.org/10.1172/JCI66778