Liver-directed gene-based therapies for inborn errors of metabolism.

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作者： Pasquale,Piccolo ^[1] ; Alessandro,Rossi ^[2] ; Nicola,Brunetti-Pierri ^[2]

作者单位： Telethon Institute of Genetics and Medicine , Pozzuoli, Italy. ^[1] Department of Translational Medicine, Federico II University of Naples , Naples, Italy. ^[2]

关键词 Liver-directed gene therapy adeno-associated virus biochemical genetics clinical trials gene therapy inborn errors of metabolism

主题词依赖病毒(Dependovirus);遗传载体(Genetic Vectors);人类(Humans);肝(Liver);代谢缺陷, 先天性(Metabolism, Inborn Errors)

DOI 10.1080/14712598.2020.1817375

PMID 32880494

发布时间 2021-11-25

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Expert opinion on biological therapy

2021年21卷2期

229-240页

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Liver-directed gene-based therapies for inborn errors of metabolism.

Expert opinion on biological therapy

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Liver-directed gene-based therapies for inborn errors of metabolism.

Expert opinion on biological therapy

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