Machine-guided design of cell-type-targeting cis-regulatory elements.

ABSTRACT: Cis-regulatory elements (CREs) control gene expression, orchestrating tissue identity, developmental timing and stimulus responses, which collectively define the thousands of unique cell types in the body^1-3. While there is great potential for strategically incorporating CREs in therapeutic or biotechnology applications that require tissue specificity, there is no guarantee that an optimal CRE for these intended purposes has arisen naturally. Here we present a platform to engineer and validate synthetic CREs capable of driving gene expression with programmed cell-type specificity. We take advantage of innovations in deep neural network modelling of CRE activity across three cell types, efficient in silico optimization and massively parallel reporter assays to design and empirica

SUBMITTER: Gosai SJ 

PROVIDER: S-EPMC11525185 | biostudies-literature | 2024 Oct

REPOSITORIES: biostudies-literature