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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...
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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2022
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Materias: | |
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 |
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