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State-of-the-Art 2019 on Gene Therapy for Phenylketonuria

Phenylketonuria (PKU) is considered to be a paradigm for a monogenic metabolic disorder but was never thought to be a primary application for human gene therapy due to established alternative treatment. However, somewhat unanticipated improvement in neuropsychiatric outcome upon long-term treatment...

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Foilsithe in:Hum Gene Ther
Main Authors: Grisch-Chan, Hiu Man, Schwank, Gerald, Harding, Cary O., Thöny, Beat
Formáid: Artigo
Teanga:Inglês
Foilsithe: Mary Ann Liebert, Inc., publishers 2019
Ábhair:
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC6763965/
https://ncbi.nlm.nih.gov/pubmed/31364419
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2019.111
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