17-Sep-2025
Could CRISPR could fix this severe childhood brain disorder?
University of California - San FranciscoPeer-Reviewed Publication
When brain development gets off to a bad start, the consequences are lifelong.
One example is a condition called SCN2A haploinsufficiency, in which children are born with just one functioning copy of the SCN2A gene — instead of the normal two. They develop defects in the connections, or synapses, between some of their brain cells; they do not learn to speak; and many of them experience seizures.
- Journal
- Nature
- Funder
- NIH/National Institutes of Health, NIH/National Institutes of Health, NIH/National Institutes of Health, NIH/National Institutes of Health, NIH/National Institutes of Health, NIH/National Institutes of Health, NIH/National Institutes of Health, NIH/National Institutes of Health, NIH/National Institutes of Health, NIH/National Institutes of Health, NIH/National Institutes of Health, Simons Foundation Autism Research Initiative, Simons Foundation Autism Research Initiative, Broad Institute Target Practice Initiative, Autism 955 Science Foundation, Weill Neurohub Investigator Program, Natural Sciences and Engineering Research Council of Canada, Ford Foundation Dissertation Fellowship, Weill Foundation