Long-Term Metabolic Correction of Phenylketonuria by AAV-Delivered Phenylalanine Amino Lyase

Phenylketonuria (PKU) is an inherited metabolic disorder caused by mutation within phenylalanine hydroxylase (PAH) gene. Loss-of-function of PAH leads to accumulation of phenylalanine in the blood/body of an untreated patient, which damages the developing brain, causing severe mental retardation. Cu...

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Vydáno v:Mol Ther Methods Clin Dev
Hlavní autoři: Tao, Rui, Xiao, Lin, Zhou, Lifang, Zheng, Zhaoyue, Long, Jie, Zhou, Lixing, Tang, Minghai, Dong, Biao, Yao, Shaohua
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2020
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7733040/
https://ncbi.nlm.nih.gov/pubmed/33335942
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.12.014
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