Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH

Phenylketonuria is an inborn error of metabolism caused by loss of function of the liver-expressed enzyme phenylalanine hydroxylase and is characterized by elevated systemic phenylalanine levels that are neurotoxic. Current therapies do not address the underlying genetic disease or restore the natur...

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Publicado no:Mol Ther Methods Clin Dev
Main Authors: Ahmed, Seemin S., Rubin, Hillard, Wang, Minglun, Faulkner, Deiby, Sengooba, Arnold, Dollive, Serena N., Avila, Nancy, Ellsworth, Jeff L., Lamppu, Diana, Lobikin, Maria, Lotterhand, Jason, Adamson-Small, Laura, Wright, Teresa, Seymour, Albert, Francone, Omar L.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7118282/
https://ncbi.nlm.nih.gov/pubmed/32258219
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.03.009
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