Enhancer-targeted CRISPR-A rescues haploinsufficiency and mutant phenotypes in organoid models of autism
George T Chen, Gayatri Nair, Aubrey J. Osorio, Sandra M. Holley, Kimiya Ghassemzadeh, Yixuan Zhou, Jose Gonzalez, Jacqueline M. Martin, Congyi Lu, Neville E. Sanjana, Carlos Cepeda, 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.
bioRxiv · Mar 13, 2024 · PreprintNeuroscience