Neonatal GALT gene replacement offers metabolic and phenotypic correction through early adulthood in a rat model of classic galactosemia

Classic galactosemia (CG) results from profound deficiency of galactose‐1‐P uridylyltransferase (GALT). Despite early detection by newborn screening and lifelong dietary restriction of galactose, most patients grow to experience a range of long‐term complications. Recently, we developed and characte...

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Detalles Bibliográficos
Autores principales: Daenzer, Jennifer M. I., Rasmussen, Shauna A., Patel, Sneh, McKenna, James, Fridovich‐Keil, Judith L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2022
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8930472/
https://www.ncbi.nlm.nih.gov/pubmed/34964137
http://dx.doi.org/10.1002/jimd.12471

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8930472/
https://www.ncbi.nlm.nih.gov/pubmed/34964137
http://dx.doi.org/10.1002/jimd.12471

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