Loading...
Publication
Genetically Modulated Substrate Reduction Therapy for Sanfilippo Syndrome: Proof of Principle
| Name: | Description: | Size: | Format: | |
|---|---|---|---|---|
| 1.97 MB | Adobe PDF |
Advisor(s)
Abstract(s)
About proof of principle in genetically modulated substrate reduction therapy for Sanfilippo syndrome.
Description
Keywords
Lysosomal Storage Diseases Mucopilysaccharidoses Substrate Reduction Therapy RNAi Doenças Genéticas Genética Humana