Reprogramming the genome with CRISPR
- 2. Edward Perello Desktop Genetics Our panel Megan Palmer Stanford University Rachel Haurwitz Caribou Bio Kevin Esvelt MIT
- 3. CRISPR is a bacterial immune system Bhaya et al. 2011
- 4. How does CRISPR edit genes?
- 5. How does CRISPR edit genes?
- 6. How does CRISPR edit genes?
- 7. How does CRISPR edit genes?
- 8. What can you do with it STABLE MUTATIONS GENETIC CORRELATION ANIMAL MODELS GENE THERAPIES
- 9. Incredible things done in health Degenerative blindness Custom cancer models Humanization of heart valves Swine fever resistance HIV receptor protein disabled Immunomodulation Gene editing has cured cancer and HIV
- 10. CRISPR is used across diverse markets and applications THERAPEUTICS AG-BIOTECHBIOLOGICAL RESEARCH INDUSTRIAL BIOTECH • Gene and cell therapies • Antimicrobials • Animal health • Precision breeding • Novel trait • generation • Crop genetics • Cell engineering • Animal models • Phenotypic screening • Genomics • Microbial fermentation • Therapeutic bioproduction
- 11. Agricultural application areas • The CRISPR technology can be used to better understand basic plant genetics • Breeding to develop crops with improved performance: • Drought tolerance • Pathogen resistance • Develop new crops 5-7 years faster • End product is identical to plants generated by traditional breeding
- 12. Industrial application areas • “Bioproduction” and “industrial fermentation” – cells manufacture a product • EG. detergent enzymes, ethanol, pharmaceutical drugs, and vaccines • CRISPR systems can be used to make production cells better factories
- 13. Genome Esvelt KM, Smidler AL, Catteruccia F, Church GM (2014) eLife CRISPR + Altered Gene How does a gene drive work?
- 14. Guide Cas9 Genome Esvelt KM, Smidler AL, Catteruccia F, Church GM (2014) eLife CRISPR + Altered Gene How does a gene drive work?
- 15. Genome Guide Cas9 CRISPR + Altered Gene How does a gene drive work? Esvelt KM, Smidler AL, Catteruccia F, Church GM (2014) eLife
- 16. How does a gene drive work? Esvelt KM, Smidler AL, Catteruccia F, Church GM (2014) eLife
- 17. How does a gene drive work?
- 18. Esvelt KM, Smidler AL, Catteruccia F, Church GM (2014) eLife How does a gene drive work?
- 19. Gantz VM, Bier E (2015) Science DiCarlo J, Chavez A, Dietz S, Esvelt KM, Church GM (2015) Nature Biotechnology Gantz VM, Jasinskiene N, Tatarenkova O, Fazekas A, Macis VM, Bier E, James AA (2015) PNAS Hammond A et al (2016) Nature Biotechnology Esvelt KM, Smidler AL, Catteruccia F, Church GM (2014) eLife CRISPR “drives” altered gene through population
- 20. Esvelt KM, Smidler AL, Catteruccia F, Church GM (2014) eLife Case-by-case evaluation of interventions
- 21. - Scientists are responsible for all consequences Safeguards Transparency - Collective scrutiny > regulation Community Guidance and Ownership - Make science responsive to the will of the people - Model: open-source software Responsive Science Unilateral ecological engineering challenges our democratic ideals
- 22. How might we ensure that the development of biotechnology is (overwhelmingly) constructive…? …or at the very least, not destructive? Going forward
- 23. Dual-use biotech posing policy puzzles While the headlines suggest CRISPR will both save & destroy the world, reality is more nuanced. Gene Drives Take Flight? Brew Your Own Heroin? Editing Human Genomes?
- 24. Rapid advances can pose risks due to pacing Need to couple innovation in gene editing with innovations in risk governance (technical + social)
- 25. Strategies for managing biological risk Scientific, Industry Norms Govt Regulations Intl Treaties Response Capacities
- 26. New challenges & strategies to manage risks Global Communities & Lower Barriers to Entry Means Leadership, Science-Policy Partnerships Are Essential