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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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Détails bibliographiques
Auteurs principaux: Rebuffat, Alexandre, Harding, Cary O., Ding, Zhaobing, Thöny, Beat
Format: Artigo
Langue:Inglês
Publié: Mary Ann Liebert, Inc. 2010
Sujets:
Accès en ligne: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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