guides de and uides...’ rawing screen’ ct ’OK’ NC STATE UNIVERSITY Innovating using CRISPR-Cas systems NC State | CALS | Food Science | CRISPR lab | Rodolphe Barrangou 2 NC STATE UNIVERSITY •! The right... •! •! •! •! Topic Time Place Business Opportunities CRISPR technology 3 NC STATE UNIVERSITY •! Genome editing and genetic engineering ATCG! ::::! TAGC! •! CRISPR = molecular scalpel •! Advantages - Programmable - Specific - Transferable - Efficient - Precise - Affordable - Quick - Multiplexable •! Gaps & needs - Large / packaging and delivery REC sgRNA Target DNA - PAM-dependent targeting - Off target cleavage - Various efficiencies •! Applications^3AgBtechMed^3EdTxMod CRISPR technology HNH PAM RuvC NGG NUC Cas9 4 NC STATE UNIVERSITY •! CRISPR literature 250 Publications •! Disruptive technology 300 200 150 100 50 •! Visible topic 0 2007 2008 2009 2010 2011 •! CRISPR has already supplanted incumbent technologies CRISPR | a compelling topic 2012 2013 5 NC STATE UNIVERSITY •! 2013: The year of the CRISPR Craze •! News & media coverage 2014| a compelling time 6 NC STATE UNIVERSITY •! •! •! •! •! Commercial products Business interest trifecta Translational potential Gene therapy momentum large, mid-size, start ups, Across the board VCs, non-profits sgRNAs | a compelling business 7 NC STATE UNIVERSITY •! •! •! •! •! •! Publications and citations IP portfolio & TTO Ambitious plans for CRISPR Core competencies & expert teams Science, technology, applications Amazing collaborative network NC State | a compelling place 8 NC STATE UNIVERSITY Lab research foci sgRNA REC CRISPR guide RNAs HNH PAM NGG Guide design and composition optimization Guide specificity and efficiency Cas9 nucleases Identify orthogonal systems (Cas9:guideRNA pairs) Develop a panel of diverse systems (Cas9, gRNAs, PAMs) Validate and disseminate N-Gen GRAS systems Outstanding issues Delivery methods Safety, toxicity, regulatory assessment RuvC NUC Cas9 9 NC STATE UNIVERSITY 1.! High-resolution bacterial genotyping (strains an populations) 2.! Harnessing CRISPR for phage resistance (dairy starter cultures) 3.! Exploiting CRISPR to limit plasmid transfer (limit uptake and dissemination of ABR) 4.! Genome evolution studies (host and viruses) 5.! Genome editing (any genome, any site, any way) 6.! Cell line development (model disease: liver cancer, BRCA1) 7.! Transcriptional control (CRISPRi/on) 8.! Chromosome imaging (EGFP) 9.! Gene Therapy (Dmd, HIV-1, cataract, tyrosinemia, beta-thalassemia, cystic fibrosis) 10.! N-gen antimicrobials and targeted killing 11.! Gene Drives (malaria-mosquito) 12.! Genome simplification and integrity enhancement Applications (chronologically) 10 NC STATE UNIVERSITY •! The right... •! •! •! •! •! •! •! Topic (CRISPR technology) Time (CRISPR Craze) Place (CRISPR@NCState) Team (CRISPR lab, CRISPR experts) Business Opportunities (CRISPR-Cas9) Deliverables (CRISPRguides, GRAS Cas9) Investment (IP, expertise, enabling technology) Chromium 24 Iodine 53 Sulfur 16 Praseodymium 59 51.996 126.90 32.065 140.91 Cr I S Pr All ingredients are in place
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