Lysosomal storage disease-associated neuropathy: Targeting stable nucleic acid lipid particle (snalp)-formulated sirnas to the brain as a therapeutic approach
More than two thirds of Lysosomal Storage Diseases (LSDs) present central nervous system involvement. Nevertheless, only one of the currently approved therapies has an impact on neuropathology. Therefore, alternative approaches are under development, either addressing the underlying enzymatic defect...
| Main Author: | |
|---|---|
| Other Authors: | , , , , , , |
| Format: | article |
| Language: | eng |
| Published: |
2020
|
| Subjects: | |
| Online Access: | https://hdl.handle.net/10216/143535 |
| Country: | Portugal |
| Oai: | oai:repositorio-aberto.up.pt:10216/143535 |