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Starting the conversation on gene therapy for phenylketonuria: Current perspectives of patients, caregivers, and advocates
Phenylketonuria (PKU) is a rare genetic condition caused by inborn error(s) in the gene for the enzyme phenylalanine hydroxylase. Resulting loss of phenylalanine (Phe) metabolism requires strict dietary therapy and/or medication to prevent toxic accumulation of Phe. Novel investigational therapies,...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Elsevier
2022
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9248220/ https://www.ncbi.nlm.nih.gov/pubmed/35782620 http://dx.doi.org/10.1016/j.ymgmr.2022.100855 |