Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery
Mucopolysaccharidosis type II (MPSII; Hunter syndrome) is a lysosomal storage disorder caused by a deficiency in the enzyme iduronate 2-sulfatase (IDS). At present, the therapeutic approaches for MPSII are enzyme replacement therapy and bone marrow transplantation, although these therapies have some...
Salvato in:
| Autori principali: | , , , , , , , |
|---|---|
| Natura: | Artigo |
| Lingua: | inglese |
| Pubblicazione: |
2006
|
| Accesso online: | https://doi.org/10.1093/hmg/ddl038 https://academic.oup.com/hmg/article-pdf/15/7/1225/17243398/ddl038.pdf |
| Tags: |
Aggiungi Tag
Nessun Tag, puoi essere il primo ad aggiungerne!!
|