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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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Hlavní autoři: Rebuffat, Alexandre, Harding, Cary O., Ding, Zhaobing, Thöny, Beat
Médium: Artigo
Jazyk:Inglês
Vydáno: Mary Ann Liebert, Inc. 2010
Témata:
On-line přístup: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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