טוען...
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...
שמור ב:
| הוצא לאור ב: | Mol Ther Methods Clin Dev |
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| Main Authors: | , , , , , , |
| פורמט: | Artigo |
| שפה: | Inglês |
| יצא לאור: |
American Society of Gene & Cell Therapy
2019
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| נושאים: | |
| גישה מקוונת: | 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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