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Novel mRNA-Based Therapy Reduces Toxic Galactose Metabolites and Overcomes Galactose Sensitivity in a Mouse Model of Classic Galactosemia
Classic galactosemia (CG) is a potentially lethal inborn error of galactose metabolism that results from deleterious mutations in the human galactose-1 phosphate uridylyltransferase (GALT) gene. Previously, we constructed a GalT(−/−) (GalT-deficient) mouse model that exhibits galactose sensitivity i...
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| Опубликовано в: : | Mol Ther |
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| Главные авторы: | , , , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
American Society of Gene & Cell Therapy
2020
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6952165/ https://ncbi.nlm.nih.gov/pubmed/31604675 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.09.018 |
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