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,...

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Detalles Bibliográficos
Autores principales: Regier, Debra S., Bąk, Agata, Bausell, Heather, O'Reilly, Emer, Cowsert, Lex M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Research Paper
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

Internet

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

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