Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9

Methylmalonic acidemia (MMA) is a severe and potentially lethal autosomal recessive inborn error of metabolism most frequently caused by mutations in the methylmalonyl-CoA mutase (MMUT) gene. Proof-of-concept adeno-associated virus (AAV) gene therapy studies using mouse models of MMA have demonstrat...

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Detalles Bibliográficos
Autores principales: Chandler, Randy J., Di Pasquale, Giovanni, Sloan, Jennifer L., McCoy, Samantha, Hubbard, Brandon T., Kilts, Tina M., Manoli, Irini, Chiorini, John A., Venditti, Charles P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9490190/
https://www.ncbi.nlm.nih.gov/pubmed/36186952
http://dx.doi.org/10.1016/j.omtm.2022.09.001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9490190/
https://www.ncbi.nlm.nih.gov/pubmed/36186952
http://dx.doi.org/10.1016/j.omtm.2022.09.001

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