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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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Bibliografische gegevens
Hoofdauteurs: Rebuffat, Alexandre, Harding, Cary O., Ding, Zhaobing, Thöny, Beat
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Mary Ann Liebert, Inc. 2010
Onderwerpen:
Online toegang: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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