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...

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Detalles Bibliográficos
Autores principales: May, Francis J., Head, PamelaSara E., Venturoni, Leah E., Chandler, Randy J., Venditti, Charles P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
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

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