Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia

Methylmalonic acidemia (MMA) is an inborn error of metabolism mostly caused by mutations in the mitochondrial methylmalonyl-CoA mutase gene (MMUT). MMA patients suffer from frequent episodes of metabolic decompensation, which can be life threatening. To mimic both the dietary restrictions and metabo...

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Detalles Bibliográficos
Autores principales: Zhang, Shengwen, Bastille, Amy, Gordo, Susana, Ramesh, Nikhil, Vora, Jenisha, McCarthy, Elizabeth, Zhang, Xiaohan, Frank, Dylan, Ko, Chih-Wei, Wu, Carmen, Walsh, Noel, Amarwani, Shreya, Liao, Jing, Xiong, Qiang, Drouin, Lauren, Hebben, Matthias, Chiang, Kyle, Chau, B. Nelson
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2022
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9488783/
https://www.ncbi.nlm.nih.gov/pubmed/36126056
http://dx.doi.org/10.1371/journal.pone.0274774

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