Future Therapies in Galactosaemia
Aug 12, 2022•39 min
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Episode description
The podcast welcomes Professor Maria Estela Rubio-Gozalbo and welcomes back Professor Judy Fridovich-Keil to talk mRNA therapy, gene therapy and disease models in classic galactosemia.
Novel mRNA therapy restores GALT protein and enzyme activity in a zebrafish model of classic galactosemia
Britt Delnoy, et al
https://doi.org/10.1002/jimd.12512
Neonatal GALT gene replacement offers metabolic and phenotypic correction through early adulthood in a rat model of classic galactosemia
Jennifer M. I. Daenzer, et al
https://doi.org/10.1002/jimd.12471
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