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Comparison of Adeno-Associated Virus Pseudotype 1, 2, and 8 Vectors Administered by Intramuscular Injection in the Treatment of Murine Phenylketonuria

Phenylketonuria (PKU) is caused by hepatic phenylalanine hydroxylase (PAH) deficiency and is associated with systemic accumulation of phenylalanine (Phe). Previously we demonstrated correction of murine PKU after intravenous injection of a recombinant type 2 adeno-associated viral vector pseudotyped...

詳細記述

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書誌詳細
主要な著者: Rebuffat, Alexandre, Harding, Cary O., Ding, Zhaobing, Thöny, Beat
フォーマット: Artigo
言語:Inglês
出版事項: Mary Ann Liebert, Inc. 2010
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC2865356/
https://ncbi.nlm.nih.gov/pubmed/19916803
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2009.127
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