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A novel Pah-exon1 deleted murine model of phenylalanine hydroxylase (PAH) deficiency
Phenylalanine hydroxylase (PAH) deficiency, colloquially known as phenylketonuria (PKU), is among the most common inborn errors of metabolism and in the past decade has become a target for the development of novel therapeutics such as gene therapy. PAH deficient mouse models have been key to new tre...
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| Publicado en: | Mol Genet Metab |
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| Autores principales: | , , , , , , |
| Formato: | Artigo |
| Lenguaje: | Inglês |
| Publicado: |
2020
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| Materias: | |
| Acceso en línea: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8173763/ https://ncbi.nlm.nih.gov/pubmed/33051130 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2020.09.005 |
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