Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia

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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Bibliographic Details
Main Authors: Francis J. May, PamelaSara E. Head, Leah E. Venturoni, Randy J. Chandler, Charles P. Venditti
Format: Article
Language:English
Published: Elsevier 2021-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
MMA
methylmalonic acidemia
AAV
PHP.eB
CaMKII
gene therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121000711

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http://www.sciencedirect.com/science/article/pii/S2329050121000711

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