Award Details:
Award Type: Catalyst
Award #: C-096
Proposal Title: Engineering Human Protein-Based mRNA Translational Controllers for Therapeutics
PI(s): Soroush Tahmasebi (UIC) and Bryan Dickinson (UChicago)
Award Amount: $250,000.00
Application Cycle: Round 28 (Fall 2019)
Award Start Date: April 1, 2020
Award End Date: March 31, 2022
Abstract:
RNA-targeting genetic therapies hold great promise for treating human diseases, especially those for which DNA-based gene therapies cannot be used, due to either lack of efficacy or safety concerns. We have recently developed a programmable RNA manipulation strategy, known as CIRTS that relies on CRISPR- Cas system. CIRTS overcame two substantial obstacles of CRISPR-Cas systems for applications as human therapeutics: their large size and microbial origin. CIRTS-based systems are small (improving delivery) and fully humanized (avoiding immune rejection). In this proposal we aim to tailor the CIRTS technology to specifically target mRNA translation, which will open up exciting therapeutic opportunities for enhancing or suppressing protein production without genetic alterations to the cell. We will engineer a new family of translational controllers built around the CIRTS technology. After optimizing the systems through iterative cycles of design and testing, we will deploy the most effective systems in preclinical models of cell growth and repair.