Dr. David Benjamin Cox
M.D. Candidate
Harvard-MIT Program in Health Sciences and Technology
Harvard Medical School, USA

Date: Tuesday, 18-December-2018
Time: 11:00 a.m.
Venue: Seminar Room 1A, G/F, Jockey Club Building for Interdisciplinary Research
5 Sassoon Road, Pokfulam, Hong Kong​

Summary:
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.

ALL ARE WELCOME