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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...

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Main Authors: Rebuffat, Alexandre, Harding, Cary O., Ding, Zhaobing, Thöny, Beat
Formato: Artigo
Idioma:Inglês
Publicado: Mary Ann Liebert, Inc. 2010
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Acceso en liña: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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