NeurosciencebioRxivPreprint
Enhancer-targeted CRISPR-A rescues haploinsufficiency and mutant phenotypes in organoid models of autism
George T Chen, Gayatri Nair, Aubrey J. Osorio…·Daniel H. Geschwind
This study uses CRISPR-A to enhance expression of CHD8 and SCN2A in organoids, rescuing haploinsufficiency and associated phenotypes in ASD models.
9 citations