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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...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
John Wiley & Sons, Inc.
2022
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| Materias: | |
| 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 |