Scalable, cell type-selective, AAV-based in vivo CRISPR screening in the mouse brain.

Biswarathan RamaniIndigo V L RoseAndrew PanRuilin TianKeran MaJorge J PalopMartin Edward Kampmann

Published in: bioRxiv : the preprint server for biology (2023)

CRISPR-based genetic screening directly in mammalian tissues in vivo is challenging due to the need for scalable, cell-type selective delivery and recovery of guide RNA libraries. We developed an in vivo adeno-associated virus-based and Cre recombinase-dependent workflow for cell type-selective CRISPR interference screening in mouse tissues. We demonstrate the power of this approach by identifying neuron-essential genes in the mouse brain using a library targeting over 2000 genes.

Keyphrases

genome wide
genome editing
crispr cas
dna methylation
gene expression
copy number
gene therapy
drug delivery
electronic health record