Targeting RNA opens therapeutic avenues for Timothy syndrome

Silvia Velasco ()

Nature, 2024, vol. 628, issue 8009, 730-732

Abstract: A therapeutic strategy that alters gene expression in a rare and severe neurodevelopmental condition has been tested in stem-cell-based models of the disease, and has been shown to correct genetic and cellular defects.

Keywords: Autism spectrum disorders; Gene therapy; Therapeutics; Stem cells (search for similar items in EconPapers)
Date: 2024
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DOI: 10.1038/d41586-024-00911-1

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