In vivo genome editing at the albumin locus to treat methylmalonic acidemia

Methylmalonic acidemia (MMA) is a metabolic disorder most commonly caused by mutations in the methylmalonyl-CoA mutase (MMUT) gene. Although adeno-associated viral (AAV) gene therapy has been effective at correcting the disease phenotype in MMA mouse models, clinical translation may be impaired by l...

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Detalles Bibliográficos
Autores principales: Schneller, Jessica L., Lee, Ciaran M., Venturoni, Leah E., Chandler, Randy J., Li, Ang, Myung, Sangho, Cradick, Thomas J., Hurley, Ayrea E., Lagor, William R., Bao, Gang, 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/PMC8634044/
https://www.ncbi.nlm.nih.gov/pubmed/34901307
http://dx.doi.org/10.1016/j.omtm.2021.11.004

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