Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia

Propionic acidemia (PA) is rare autosomal recessive metabolic disorder caused by defects in the mitochondrially localized enzyme propionyl-coenzyme A (CoA) carboxylase. Patients with PA can suffer from lethal metabolic decompensation and cardiomyopathy despite current medical management, which has l...

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Detalles Bibliográficos
Autores principales: Chandler, Randy J., Di Pasquale, Giovanni, Choi, Eun-Young, Chang, David, Smith, Stephanie N., Sloan, Jennifer L., Hoffmann, Victoria, Li, Lina, Chiorini, John A., Venditti, Charles P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10512014/
https://www.ncbi.nlm.nih.gov/pubmed/37746248
http://dx.doi.org/10.1016/j.omtm.2023.06.008

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