A carregar...
Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia
Methylmalonic acidemia (MMA) is a severe metabolic disorder most commonly caused by a mutation in the methylmalonyl-CoA mutase (MMUT) gene. Patients with MMA experience multisystemic disease manifestations and remain at risk for neurological disease progression, even after liver transplantation. The...
Na minha lista:
| Publicado no: | Mol Ther Methods Clin Dev |
|---|---|
| Main Authors: | , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Gene & Cell Therapy
2021
|
| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8188058/ https://ncbi.nlm.nih.gov/pubmed/34169115 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2021.04.005 |
| Tags: |
Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!
|