Novel mRNA-Based Therapy Reduces Toxic Galactose Metabolites and Overcomes Galactose Sensitivity in a Mouse Model of Classic Galactosemia-Reference-Cited by-同舟云学术

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Novel mRNA-Based Therapy Reduces Toxic Galactose Metabolites and Overcomes Galactose Sensitivity in a Mouse Model of Classic Galactosemia

Published:2020-01 Issue:1 Volume:28 Page:304-312
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Balakrishnan Bijina,An Ding,Nguyen Vi,DeAntonis Christine,Martini Paolo G.V.,Lai Kent

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference56 articles.

1. Congenital galactosemia, a single enzymatic block in galactose metabolism;Isselbacher;Science,1956

2. Galactosemia: the good, the bad, and the unknown;Fridovich-Keil;J. Cell. Physiol.,2006

3. Classical galactosemia and mutations at the galactose-1-phosphate uridyl transferase (GALT) gene;Tyfield;Hum. Mutat.,1999

4. Disorders of Galactose Metabolism;Berry,2006

5. Disorders of galactose metabolism;Segal,1995

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