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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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| 出版年: | Hum Gene Ther |
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| 主要な著者: | , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
Mary Ann Liebert, Inc., publishers
2019
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| 主題: | |
| オンライン･アクセス: | 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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