Machine-guided design of synthetic cell type-specific 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. 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 an intended purpose has arisen naturally through evolution. Here, we present a platform to engineer and validate synthetic CREs capable of driving gene expression with programmed cell type specificity. We leverage innovations in deep neural network modeling of CRE activity across three cell types, efficient in silico optimization, and massively parallel reporter assays (MPRAs) to desi

SUBMITTER: Gosai SJ 

PROVIDER: S-EPMC10441439 | biostudies-literature | 2023 Aug

REPOSITORIES: biostudies-literature