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REPROGRAMMING THE GENOME
Edward Perello Desktop Genetics
Our panel
Megan PalmerStanford University
Rachel HaurwitzCaribou Bio
Kevin EsveltMIT
CRISPR is a bacterial immune system
Bhaya et al. 2011
How does CRISPR edit genes?
How does CRISPR edit genes?
How does CRISPR edit genes?
How does CRISPR edit genes?
What can you do with it
STABLE MUTATIONS
GENETIC CORRELATION
ANIMAL MODELS GENE THERAPIES
Incredible things done in health
Degenerative blindnessCustom cancer models
Humanization of heart valvesSwine fever resistance
HIV receptor protein disabled
Immunomodulation
Gene editing has cured cancer and HIV
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
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
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
Genome
Esvelt KM, Smidler AL, Catteruccia F, Church GM (2014) eLife
CRISPR+
Altered Gene
How does a gene drive work?
Guide
Cas9
Genome
Esvelt KM, Smidler AL, Catteruccia F, Church GM (2014) eLife
CRISPR+
Altered Gene
How does a gene drive work?
Genome
Guide
Cas9CRISPR
+ Altered Gene
How does a gene drive work?
Esvelt KM, Smidler AL, Catteruccia F, Church GM (2014) eLife
How does a gene drive work?
Esvelt KM, Smidler AL, Catteruccia F, Church GM (2014) eLife
How does a gene drive work?
Esvelt KM, Smidler AL, Catteruccia F, Church GM (2014) eLife
How does a gene drive work?
Gantz VM, Bier E (2015) ScienceDiCarlo 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) PNASHammond A et al (2016) Nature Biotechnology
Esvelt KM, Smidler AL, Catteruccia F, Church GM (2014) eLife
CRISPR “drives” altered gene through population
Esvelt KM, Smidler AL, Catteruccia F, Church GM (2014) eLife
Case-by-case evaluation of interventions
- Scientists are responsible for all consequencesSafeguards
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
How might we ensure that the development of biotechnology is (overwhelmingly) constructive…?
…or at the very least, not destructive?
Going forward
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?
Rapid advances can pose risks due to pacing
Need to couple innovation in gene editing with innovations
in risk governance (technical + social)
Strategies for managing biological risk
Scientific, Industry Norms
Govt Regulations
Intl Treaties
Response Capacities
New challenges & strategies to manage risks
Global Communities & Lower Barriers to Entry
Means Leadership,Science-Policy PartnershipsAre Essential
