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AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria
Phenylketonuria (PKU) due to recessively inherited phenylalanine hydroxylase (PAH) deficiency results in hyperphenylalaninemia, which is toxic to the central nervous system. Restriction of dietary phenylalanine intake remains the standard of PKU care and prevents the major neurologic manifestations...
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| Publicado no: | Mol Ther Methods Clin Dev |
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| Main Authors: | , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Gene & Cell Therapy
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6962637/ https://ncbi.nlm.nih.gov/pubmed/31970201 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.12.004 |
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