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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...
Enregistré dans:
| Publié dans: | Mol Ther Methods Clin Dev |
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| Auteurs principaux: | , , , , |
| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
American Society of Gene & Cell Therapy
2021
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| Sujets: | |
| Accès en ligne: | 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 |
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