CRISPR approach offers hope for severe childhood brain disorder

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.

This article was originally published on MedicalXpress.com

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