Development of an mRNA replacement therapy for phenylketonuria

Phenylketonuria (PKU) is an inborn error caused by deficiencies in phenylalanine (Phe) metabolism. Mutations in the phenylalanine hydroxylase (PAH) gene are the main cause of the disease whose signature hallmarks of toxically elevated levels of Phe accumulation in plasma and organs such as the brain...

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Detalles Bibliográficos
Autores principales: Perez-Garcia, Carlos G., Diaz-Trelles, Ramon, Vega, Jerel Boyd, Bao, Yanjie, Sablad, Marciano, Limphong, Patty, Chikamatsu, Simon, Yu, Hailong, Taylor, Wendy, Karmali, Priya P., Tachikawa, Kiyoshi, Chivukula, Padmanabh
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/PMC8933640/
https://www.ncbi.nlm.nih.gov/pubmed/35356682
http://dx.doi.org/10.1016/j.omtn.2022.02.020

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8933640/
https://www.ncbi.nlm.nih.gov/pubmed/35356682
http://dx.doi.org/10.1016/j.omtn.2022.02.020

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