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Poster Presentation: Engineering Cell-Type-Specific Promoters for In Vivo Cell Therapies

08 Sept 2026
Next Gen Vector Engineering & Delivery
In vivo cell therapy reprograms patient cells directly within the body demands regulatory elements that restrict transgene expression to target lineages. We supported our client
to develop compact promoters that are strong in T and NK cells yet silent in HSCs and B cells. Combining RNA-seq/ATAC-seq bioinformatics, a 36,000-member synthetic
TFBS library screened by MPRA, and native element discovery, we identified specific-but-weak synthetic hits and strong-but-nonspecific native promoters. Mutagenesis pinpointed HSC- and B-cell repressive elements, and recombining native with synthetic components yielded promoters with both high strength and tight T/NK specificity, enabling precise in vivo targeting.
Industry Expert
Doug Hattendorf, Director, Business Development & Alliance Management - GeneFab

GENEFAB