Article

Large-scale discovery of neural enhancers for cis-regulation therapies

2025-11-05

Abstract excerpt

CRISPR-based gene activation (CRISPRa) has emerged as a promising therapeutic approach for neurodevelopmental disorders (NDD) caused by haploinsufficiency. However, scaling this cis -regulatory therapy (CRT) paradigm requires pinpointing which candidate cis -regulatory elements (cCREs) are active in human neurons, and which can be targeted with CRISPRa to yield specific and therapeutic levels of target gene upre...

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Literature Corpus work: 3a4f8df6-a172-505a-bcde-e5f21c60f11d
DOI: 10.1101/2025.11.04.686611

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