2020-04-23T15:59:27+08:002018-12-18|Seminars and Workshops|

Talk title RNA editing with CRISPR-Cas13
Speaker Dr. David COX
PhD recipient
Harvard-MIT Program in Health Sciences and Technology, Harvard Medical School
Date & Time 17 December 2018 (Mon)
Venue Room G004, E12 Building (University of Macau)
Abstract Nucleic acid editing holds promise for treating genetic disease, particularly at the RNA level, where disease-relevant sequences can be rescued to yield functional protein products. Type VI CRISPR-Cas systems contain the programmable single-effector RNA-guided RNases Cas13. Here, we profile Type VI systems to engineer a Cas13 ortholog capable of robust knockdown and demonstrate RNA editing by using catalytically-inactive Cas13 (dCas13) to direct adenosine to inosine deaminase activity by ADAR2 to transcripts in mammalian cells. This system, referred to as RNA Editing for Programmable A to I Replacement (REPAIR), which has no strict sequence constraints, can be used to edit full-length transcripts containing pathogenic mutations. We further engineer this system to create a high specificity variant and minimize the system to facilitate viral delivery. REPAIR presents a promising RNA editing platform with broad applicability for research, therapeutics, and biotechnology.