A carregar...
Overcoming Limitations Inherent in Sulfamidase to Improve Mucopolysaccharidosis IIIA Gene Therapy
Sulfamidase (SGSH) deficiency causes mucopolysaccharidosis type IIIA (MPS IIIA), a lysosomal storage disease (LSD) that affects the CNS. In earlier work in LSD mice and dog models, we exploited the utility of adeno-associated viruses (AAVs) to transduce brain ventricular lining cells (ependyma) for...
Na minha lista:
| Publicado no: | Mol Ther |
|---|---|
| Main Authors: | , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Gene & Cell Therapy
2018
|
| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6079371/ https://ncbi.nlm.nih.gov/pubmed/29503202 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.01.010 |
| Tags: |
Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!
|