N6-Room G001, Administration Building, Graduate School
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FHS Seminar Series - "RNA editing with CRISPR-Cas13" - Dr. David Cox
Seminar / Lecture
Faculty of Health Sciences
17 Dezembro 2018
E12-G004, Faculty of Health Sciences
Dr. Cox received his M.D. and PhD from Harvard University-MIT Program in Health Sciences and Technology Harvard Medical School. His research is mainly on genome editing and he has already applied serval international patents.
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.