Tunable, self-contained gene dosage control via proteolytic cleavage of CRISPR-Cas systems
Noa Katz,
Connie An,
Yu-Ju Lee
et al.
Abstract:SummaryGene therapy holds great therapeutic potential. Yet, controlling cargo expression in single cells is limited due to the variability of delivery methods. We implement an incoherent feedforward loop based on proteolytic cleavage of CRISPR-Cas activation or inhibition systems to reduce gene expression variability against the variability of vector delivery. We demonstrate dosage control for activation and inhibition, post-delivery tuning, and RNA-based delivery, for a genome-integrated marker. We then targe… Show more
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