Fragile X syndrome is a genetic disorder and the leading cause of intellectual disability and autism. There is no treatment. In this episode, Kellen discusses recent gene therapy advances for treating Fragile X syndrome. The article highlighted in this episode was written by X. Shawn Liu and colleagues from Rudolf Jaenisch’s lab at the Whitehead Institute and is titled “Rescue of Fragile X Syndrome Neurons by DNA Methylation Editing of the FMR1 Gene”.

Paper: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6375087/

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