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Future Therapies in Galactosaemia

EPISODE · Aug 12, 2022 · 39 MIN

Future Therapies in Galactosaemia

from JIMD Podcasts · host Journal of Inherited Metabolic Disease

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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Future Therapies in Galactosaemia

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