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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...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8188058/ https://www.ncbi.nlm.nih.gov/pubmed/34169115 http://dx.doi.org/10.1016/j.omtm.2021.04.005 |