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

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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Bibliographic Details
Main Authors: Rui Tao, Lin Xiao, Lifang Zhou, Zhaoyue Zheng, Jie Long, Lixing Zhou, Minghai Tang, Biao Dong, Shaohua Yao
Format: Article
Language:English
Published: Elsevier 2020-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
phenylketonuria
adeno-associated virus
phenylalanine amino lyase
gene therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S232905012030005X

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http://www.sciencedirect.com/science/article/pii/S232905012030005X

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