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Thank you for the post HHMI. This study is a key first step toward finding a cure for Dravet Syndrome.
Dravet syndrome is a rare, often fatal form of childhood epilepsy. New research from David Liu (Harvard University, Broad Institute of MIT and Harvard) & collaborators found that a single dose of base editing can correct its underlying mutation in mice, dramatically reducing seizures & extending survival. “This study gives us hope that base editing could be an effective approach for durably correcting the underlying cause of Dravet syndrome in patients,” Liu says. bit.ly/4nLvIAM

Image: Mouse neurons courtesy of Christophe Leterrier, NeuroCyto Lab, INP, Marseille, France, via NIH BRAIN Initiative.

Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study Study in mice shows that base editing can correct the genetic mutation behind Dravet syndrome