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PRESCOUTER 1
The presentation will begin shortly...
WHILE YOU WAIT:
 Stay up to date with industry disruptors and R&D eve...
PRESCOUTER 2
PRESCOUTER
CRISPR:
Opportunities and Challenges Webinar
Your Personal Research Assistant
PRESCOUTER 3
Webinar Host
PRESCOUTER
 Medical Lead Scientist at PreScouter, Inc.
 Ph.D. Biophysical Sciences from the Un...
PRESCOUTER 4
What is CRISPR/Cas?
PRESCOUTER
 CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats): repeatin...
PRESCOUTER 5
What is CRISPR/Cas?
PRESCOUTER
 CRISPR/Cas: form of acquired immunity in prokaryotes that confers resistance...
PRESCOUTER 6
How the CRISPR/Cas9 system works
PRESCOUTER
 sgRNA (single guide RNA): synthetic RNA molecule that contains ...
PRESCOUTER 7
How the CRISPR/Cas9 system works
PRESCOUTER
 Cas9: a Cas endonuclease protein that can cleave almost any DNA...
PRESCOUTER 8
How the CRISPR/Cas9 system works
PRESCOUTER
PRESCOUTER 9
How the CRISPR/Cas9 system works
PRESCOUTER
PRESCOUTER 10
CRISPR Timeline
PRESCOUTER
1987
2002
2005
2006
2007
2010
2012
2013
2014
2015
2016
2017
CRISPR repeats first ...
PRESCOUTER
CRISPR repeats first observed in bacterial genomes
CRISPR elements and associated genes identified and named
CR...
PRESCOUTER 12
CRISPR Timeline
PRESCOUTER
1987
2002
2005
2006
2007
2010
2012
2013
2014
2015
2016
2017
CRISPR repeats first ...
PRESCOUTER 13
CRISPR Timeline
PRESCOUTER
1987
2002
2005
2006
2007
2010
2012
2013
2014
2015
2016
2017
CRISPR repeats first ...
PRESCOUTER 14
CRISPR Timeline
PRESCOUTER
1987
2002
2005
2006
2007
2010
2012
2013
2014
2015
2016
2017
CRISPR repeats first ...
PRESCOUTER 15
CRISPR Timeline
PRESCOUTER
1987
2002
2005
2006
2007
2010
2012
2013
2014
2015
2016
2017
CRISPR repeats first ...
PRESCOUTER 16
CRISPR Timeline
PRESCOUTER
1987
2002
2005
2006
2007
2010
2012
2013
2014
2015
2016
2017
CRISPR repeats first ...
PRESCOUTER 17
CRISPR Timeline
PRESCOUTER
1987
2002
2005
2006
2007
2010
2012
2013
2014
2015
2016
2017
CRISPR repeats first ...
PRESCOUTER 18
Potential Applications of CRISPR
Biomedical research:
we are creating new animal models
to understand diseas...
PRESCOUTER
Your Personal Research Assistant
Technology Scouting
Market Analysis
Competitive Intelligence
Technology Evalua...
PRESCOUTER 20
3 Step Process
PRESCOUTER
STEP 1
Describe your questions Meet with your PreScouter-managed
Scholar team 2–3 ...
PRESCOUTER 21
CRISPR Panelists
PRESCOUTER
John G. Doench, Ph.D.
Associate Director of the
Genetic Perturbation
Platform at...
Questions
PRESCOUTER
What enhancements to the CRISPR/Cas9 system do
you think will be the most impactful?
1
PRESCOUTER
Of the current technical limitations facing the CRISPR technology,
which do you think are most important?
2
PRESCOUTER
What are the lessons we can learn from the obstacles faced by
CRISPR’s predecessors? In what ways is CRISPR uni...
PRESCOUTER
What are the main technical challenges of applying CRISPR to
animal models? How will CRISPR-based development o...
PRESCOUTER
5
How can CRISPR aid in research involving pluripotent stem cells?
What translational/therapeutic use cases do ...
PRESCOUTER 28
CRISPR and Pluripotent Stem Cells (PSCs)
Neuron Blood Cells Cardiomyocytes β cells Liver Cells
Modified from...
PRESCOUTER 29
Research Tools
• Create reporter line.
• Evaluate the biological function of certain genes or mutations in h...
PRESCOUTER 30
Therapeutic Application
Patient
Corrected
hPSCs
Reprogramming
Patient-specific
hPSCs
Directed
differentiatio...
PRESCOUTER
What are the greatest challenges (technical, regulatory, or ethical) to
broader applications of CRISPR within b...
PRESCOUTER
What applications of CRISPR are you most excited to see in the next
few years? What about the next 10–20 years?...
PRESCOUTER 33
Future Direction of CRISPR in Pluripotent Stem Cells
1. Gene/Mutation Specific Drugs
Patient-specific
tissue...
THANK YOU
STICK AROUND FOR OUR Q&A
PRESCOUTER
Your Personal Research Assistant
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CRISPR: Opportunities and Challenges Webinar

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CRISPR is a genome-editing technique that provides a simple yet versatile method for making targeted changes to the genome of living cells. Researchers have already applied CRISPR to reverse disease-causing mutations and to engineer pest-resistant crops.
In this CRISPR webinar, PreScouter will be joined by experts and researchers to review CRISPR's opportunities and challenges. We will touch upon key challenges, such as reducing off-target effects, as well as new advances set to overcome some of the current limitations, such as novel CRISPR systems. Additionally, we'll highlight potential first applications of the technology within medicine and agriculture.
This is a great opportunity to not only learn from experts about how this disruptive technology is changing gene editing but also ask your personal questions about CRISPR.
Moderator:
This CRISPR discussion will be moderated by Charles Wright, the Medical Project Architect at PreScouter.
Panelists:
John G. Doench, Ph.D., is Associate Director of the Genetic Perturbation Platform at the Broad Institute.
C. B. Gurumurthy (Guru), BVSC, MVSC, Ph.D. Exec. MBA, is an Assistant Professor at the Department of Developmental Neuroscience, Munroe Meyer Institute for Genetics and Rehabilitation, and he serves as the Director of UNMC Mouse Genome Engineering Core Facility at the University of Nebraska Medical Center.
Shuibing Chen is an Assistant Professor in the Department of Surgery and Biochemistry at Weill Cornell Medical College, New York.

Published in: Health & Medicine
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  • DOWNLOAD FULL BOOKS, INTO AVAILABLE FORMAT ......................................................................................................................... ......................................................................................................................... 1.DOWNLOAD FULL. PDF EBOOK here { https://tinyurl.com/y3nhqquc } ......................................................................................................................... 1.DOWNLOAD FULL. EPUB Ebook here { https://tinyurl.com/y3nhqquc } ......................................................................................................................... 1.DOWNLOAD FULL. doc Ebook here { https://tinyurl.com/y3nhqquc } ......................................................................................................................... 1.DOWNLOAD FULL. PDF EBOOK here { https://tinyurl.com/y3nhqquc } ......................................................................................................................... 1.DOWNLOAD FULL. EPUB Ebook here { https://tinyurl.com/y3nhqquc } ......................................................................................................................... 1.DOWNLOAD FULL. doc Ebook here { https://tinyurl.com/y3nhqquc } ......................................................................................................................... ......................................................................................................................... ......................................................................................................................... .............. Browse by Genre Available eBooks ......................................................................................................................... Art, Biography, Business, Chick Lit, Children's, Christian, Classics, Comics, Contemporary, Cookbooks, Crime, Ebooks, Fantasy, Fiction, Graphic Novels, Historical Fiction, History, Horror, Humor And Comedy, Manga, Memoir, Music, Mystery, Non Fiction, Paranormal, Philosophy, Poetry, Psychology, Religion, Romance, Science, Science Fiction, Self Help, Suspense, Spirituality, Sports, Thriller, Travel, Young Adult,
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CRISPR: Opportunities and Challenges Webinar

  1. 1. PRESCOUTER 1 The presentation will begin shortly... WHILE YOU WAIT:  Stay up to date with industry disruptors and R&D events by signing up for The PreScouter Newsletter on www.prescouter.com.  Check your email after the presentation for a survey and recording from this webinar.
  2. 2. PRESCOUTER 2 PRESCOUTER CRISPR: Opportunities and Challenges Webinar Your Personal Research Assistant
  3. 3. PRESCOUTER 3 Webinar Host PRESCOUTER  Medical Lead Scientist at PreScouter, Inc.  Ph.D. Biophysical Sciences from the University of Chicago Charles Wright, Ph.D.
  4. 4. PRESCOUTER 4 What is CRISPR/Cas? PRESCOUTER  CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats): repeating palindromic sequences of DNA in prokaryotes; unique spacer DNA sequences matching to foreign DNA are found between each repeat  Cas (CRISPR Associated protein): enzymes needed to identify and cut the targeted DNA sequence
  5. 5. PRESCOUTER 5 What is CRISPR/Cas? PRESCOUTER  CRISPR/Cas: form of acquired immunity in prokaryotes that confers resistance to foreign genetic elements  CRISPR/Cas9: simplified, programmable version of CRISPR/Cas modified to edit genomes
  6. 6. PRESCOUTER 6 How the CRISPR/Cas9 system works PRESCOUTER  sgRNA (single guide RNA): synthetic RNA molecule that contains the components needed to target the desired genomic DNA sequence(s) and to complex with a Cas protein
  7. 7. PRESCOUTER 7 How the CRISPR/Cas9 system works PRESCOUTER  Cas9: a Cas endonuclease protein that can cleave almost any DNA sequence complementary to its guide RNA
  8. 8. PRESCOUTER 8 How the CRISPR/Cas9 system works PRESCOUTER
  9. 9. PRESCOUTER 9 How the CRISPR/Cas9 system works PRESCOUTER
  10. 10. PRESCOUTER 10 CRISPR Timeline PRESCOUTER 1987 2002 2005 2006 2007 2010 2012 2013 2014 2015 2016 2017 CRISPR repeats first observed in bacterial genomes CRISPR elements and associated genes identified and named CRISPR spacer identified as foreign DNA CRISPR proposed to be a bacterial adaptive immune system Discovery that CRISPR/Cas imparts resistance to specific phages CRISPR/Cas identified as bacterial and archaeal immune system CRISPR/Cas9 developed as gene editing tool CRISPR/Cas9 used to edit targeted genes in both human and mouse cells First use of CRISPR/Cas9 in plants CRISPR/Cas9 used to cut HIV out of genome of infected humans cells Monkeys with CRISPR-engineered targeted mutations are born CRISPR/Cas9 used to develop virus-resistant tomato plants CRISPR/Cas9 used to edit human embryos (but many off-target effects observed) International moratorium proposed on making heritable changes to the human genome USDA determines CRISPR/Cas9 edited crops will not be regulated as GMOs First human trial to use CRISPR gene editing gets NIH approval US patent office awards key CRISPR/Cas9 patents to the Broad Institute National Academy of Sciences report outlines criteria to be met for germline editing clinical trials
  11. 11. PRESCOUTER CRISPR repeats first observed in bacterial genomes CRISPR elements and associated genes identified and named CRISPR spacer identified as foreign DNA CRISPR proposed to be a bacterial adaptive immune system Discovery that CRISPR/Cas imparts resistance to specific phages CRISPR/Cas identified as bacterial and archaeal immune system CRISPR/Cas9 developed as gene editing tool CRISPR/Cas9 used to edit targeted genes in both human and mouse cells First use of CRISPR/Cas9 in plants CRISPR/Cas9 used to cut HIV out of genome of infected humans cells Monkeys with CRISPR-engineered targeted mutations are born CRISPR/Cas9 used to develop virus-resistant tomato plants CRISPR/Cas9 used to edit human embryos (but many off-target effects observed) International moratorium proposed on making heritable changes to the human genome USDA determines CRISPR/Cas9 edited crops will not be regulated as GMOs First human trial to use CRISPR gene editing gets NIH approval US patent office awards key CRISPR/Cas9 patents to the Broad Institute National Academy of Sciences report outlines criteria to be met for germline editing clinical trials 11 CRISPR Timeline PRESCOUTER 1987 2002 2005 2006 2007 2010 2012 2013 2014 2015 2016 2017
  12. 12. PRESCOUTER 12 CRISPR Timeline PRESCOUTER 1987 2002 2005 2006 2007 2010 2012 2013 2014 2015 2016 2017 CRISPR repeats first observed in bacterial genomes CRISPR elements and associated genes identified and named CRISPR spacer identified as foreign DNA CRISPR proposed to be a bacterial adaptive immune system Discovery that CRISPR/Cas imparts resistance to specific phages CRISPR/Cas identified as bacterial and archaeal immune system CRISPR/Cas9 developed as gene editing tool CRISPR/Cas9 used to edit targeted genes in both human and mouse cells First use of CRISPR/Cas9 in plants CRISPR/Cas9 used to cut HIV out of genome of infected humans cells Monkeys with CRISPR-engineered targeted mutations are born CRISPR/Cas9 used to develop virus-resistant tomato plants CRISPR/Cas9 used to edit human embryos (but many off-target effects observed) International moratorium proposed on making heritable changes to the human genome USDA determines CRISPR/Cas9 edited crops will not be regulated as GMOs First human trial to use CRISPR gene editing gets NIH approval US patent office awards key CRISPR/Cas9 patents to the Broad Institute National Academy of Sciences report outlines criteria to be met for germline editing clinical trials
  13. 13. PRESCOUTER 13 CRISPR Timeline PRESCOUTER 1987 2002 2005 2006 2007 2010 2012 2013 2014 2015 2016 2017 CRISPR repeats first observed in bacterial genomes CRISPR elements and associated genes identified and named CRISPR spacer identified as foreign DNA CRISPR proposed to be a bacterial adaptive immune system Discovery that CRISPR/Cas imparts resistance to specific phages CRISPR/Cas identified as bacterial and archaeal immune system CRISPR/Cas9 developed as gene editing tool CRISPR/Cas9 used to edit targeted genes in both human and mouse cells First use of CRISPR/Cas9 in plants CRISPR/Cas9 used to cut HIV out of genome of infected humans cells Monkeys with CRISPR-engineered targeted mutations are born CRISPR/Cas9 used to develop virus-resistant tomato plants CRISPR/Cas9 used to edit human embryos (but many off-target effects observed) International moratorium proposed on making heritable changes to the human genome USDA determines CRISPR/Cas9 edited crops will not be regulated as GMOs First human trial to use CRISPR gene editing gets NIH approval US patent office awards key CRISPR/Cas9 patents to the Broad Institute National Academy of Sciences report outlines criteria to be met for germline editing clinical trials
  14. 14. PRESCOUTER 14 CRISPR Timeline PRESCOUTER 1987 2002 2005 2006 2007 2010 2012 2013 2014 2015 2016 2017 CRISPR repeats first observed in bacterial genomes CRISPR elements and associated genes identified and named CRISPR spacer identified as foreign DNA CRISPR proposed to be a bacterial adaptive immune system Discovery that CRISPR/Cas imparts resistance to specific phages CRISPR/Cas identified as bacterial and archaeal immune system CRISPR/Cas9 developed as gene editing tool CRISPR/Cas9 used to edit targeted genes in both human and mouse cells First use of CRISPR/Cas9 in plants CRISPR/Cas9 used to cut HIV out of genome of infected humans cells Monkeys with CRISPR-engineered targeted mutations are born CRISPR/Cas9 used to develop virus-resistant tomato plants CRISPR/Cas9 used to edit human embryos (but many off-target effects observed) International moratorium proposed on making heritable changes to the human genome USDA determines CRISPR/Cas9 edited crops will not be regulated as GMOs First human trial to use CRISPR gene editing gets NIH approval US patent office awards key CRISPR/Cas9 patents to the Broad Institute National Academy of Sciences report outlines criteria to be met for germline editing clinical trials
  15. 15. PRESCOUTER 15 CRISPR Timeline PRESCOUTER 1987 2002 2005 2006 2007 2010 2012 2013 2014 2015 2016 2017 CRISPR repeats first observed in bacterial genomes CRISPR elements and associated genes identified and named CRISPR spacer identified as foreign DNA CRISPR proposed to be a bacterial adaptive immune system Discovery that CRISPR/Cas imparts resistance to specific phages CRISPR/Cas identified as bacterial and archaeal immune system CRISPR/Cas9 developed as gene editing tool CRISPR/Cas9 used to edit targeted genes in both human and mouse cells First use of CRISPR/Cas9 in plants CRISPR/Cas9 used to cut HIV out of genome of infected humans cells Monkeys with CRISPR-engineered targeted mutations are born CRISPR/Cas9 used to develop virus-resistant tomato plants CRISPR/Cas9 used to edit human embryos (but many off-target effects observed) International moratorium proposed on making heritable changes to the human genome USDA determines CRISPR/Cas9 edited crops will not be regulated as GMOs First human trial to use CRISPR gene editing gets NIH approval US patent office awards key CRISPR/Cas9 patents to the Broad Institute National Academy of Sciences report outlines criteria to be met for germline editing clinical trials
  16. 16. PRESCOUTER 16 CRISPR Timeline PRESCOUTER 1987 2002 2005 2006 2007 2010 2012 2013 2014 2015 2016 2017 CRISPR repeats first observed in bacterial genomes CRISPR elements and associated genes identified and named CRISPR spacer identified as foreign DNA CRISPR proposed to be a bacterial adaptive immune system Discovery that CRISPR/Cas imparts resistance to specific phages CRISPR/Cas identified as bacterial and archaeal immune system CRISPR/Cas9 developed as gene editing tool CRISPR/Cas9 used to edit targeted genes in both human and mouse cells First use of CRISPR/Cas9 in plants CRISPR/Cas9 used to cut HIV out of genome of infected humans cells Monkeys with CRISPR-engineered targeted mutations are born CRISPR/Cas9 used to develop virus-resistant tomato plants CRISPR/Cas9 used to edit human embryos (but many off-target effects observed) International moratorium proposed on making heritable changes to the human genome USDA determines CRISPR/Cas9 edited crops will not be regulated as GMOs First human trial to use CRISPR gene editing gets NIH approval US patent office awards key CRISPR/Cas9 patents to the Broad Institute National Academy of Sciences report outlines criteria to be met for germline editing clinical trials
  17. 17. PRESCOUTER 17 CRISPR Timeline PRESCOUTER 1987 2002 2005 2006 2007 2010 2012 2013 2014 2015 2016 2017 CRISPR repeats first observed in bacterial genomes CRISPR elements and associated genes identified and named CRISPR spacer identified as foreign DNA CRISPR proposed to be a bacterial adaptive immune system Discovery that CRISPR/Cas imparts resistance to specific phages CRISPR/Cas identified as bacterial and archaeal immune system CRISPR/Cas9 developed as gene editing tool CRISPR/Cas9 used to edit targeted genes in both human and mouse cells First use of CRISPR/Cas9 in plants CRISPR/Cas9 used to cut HIV out of genome of infected humans cells Monkeys with CRISPR-engineered targeted mutations are born CRISPR/Cas9 used to develop virus-resistant tomato plants CRISPR/Cas9 used to edit human embryos (but many off-target effects observed) International moratorium proposed on making heritable changes to the human genome USDA determines CRISPR/Cas9 edited crops will not be regulated as GMOs First human trial to use CRISPR gene editing gets NIH approval US patent office awards key CRISPR/Cas9 patents to the Broad Institute National Academy of Sciences report outlines criteria to be met for germline editing clinical trials
  18. 18. PRESCOUTER 18 Potential Applications of CRISPR Biomedical research: we are creating new animal models to understand disease and to aid in drug discovery Agriculture: we can edit the genes of crops to make them tastier, more nutritious, pathogen-resistant, or more robust to extreme environmental conditions Gene drives: instead of modifying just a single organism, we could modify an entire species New antibiotics and antivirals: we could use this system to eradicate specific bacteria and viruses New tools to stop genetic diseases: we could edit the human genome to eliminate genetic diseases like Huntington’s disease, sickle cell anemia, or cystic fibrosis Designer humans: we might one day edit the human genome to eliminate disease or select for desirable traits
  19. 19. PRESCOUTER Your Personal Research Assistant Technology Scouting Market Analysis Competitive Intelligence Technology Evaluation 19 Technology Landscapes Start up Interviews Forecasts Market Sizing
  20. 20. PRESCOUTER 20 3 Step Process PRESCOUTER STEP 1 Describe your questions Meet with your PreScouter-managed Scholar team 2–3 times over 4–6 weeks to review what they have found STEP 2 Make smarter decisions, based on the Scholars’ report STEP 3
  21. 21. PRESCOUTER 21 CRISPR Panelists PRESCOUTER John G. Doench, Ph.D. Associate Director of the Genetic Perturbation Platform at the Broad Institute C.B. Gurumurthy, Ph.D., Exec. MBA Associate Professor at the Department of Developmental Neuroscience, Munroe Meyer Institute for Genetics and Rehabilitation Shuibing Chen, Ph.D. Assistant Professor in the Department of Surgery and Biochemistry at Weill Cornell Medical College
  22. 22. Questions
  23. 23. PRESCOUTER What enhancements to the CRISPR/Cas9 system do you think will be the most impactful? 1
  24. 24. PRESCOUTER Of the current technical limitations facing the CRISPR technology, which do you think are most important? 2
  25. 25. PRESCOUTER What are the lessons we can learn from the obstacles faced by CRISPR’s predecessors? In what ways is CRISPR unique from these systems? 3
  26. 26. PRESCOUTER What are the main technical challenges of applying CRISPR to animal models? How will CRISPR-based development of animal models allow us to create better disease models and aid in drug discovery? 4
  27. 27. PRESCOUTER 5 How can CRISPR aid in research involving pluripotent stem cells? What translational/therapeutic use cases do you foresee? What are the hurdles to achieving these applications?
  28. 28. PRESCOUTER 28 CRISPR and Pluripotent Stem Cells (PSCs) Neuron Blood Cells Cardiomyocytes β cells Liver Cells Modified from Regenerative Medicine. August 2006
  29. 29. PRESCOUTER 29 Research Tools • Create reporter line. • Evaluate the biological function of certain genes or mutations in human development and human cells. hPSCs definitive endoderm pancreatic progenitor glucose-responding cells Isogenic hPSCs
  30. 30. PRESCOUTER 30 Therapeutic Application Patient Corrected hPSCs Reprogramming Patient-specific hPSCs Directed differentiation Replacement therapy Patient Corrected tissue or organs Isogenic hPSCs Patient-specific tissues or organs Drug screening Drug candidates for precision therapy FoldChangeofINS+ CellDeathRate #of INS+ cells 0.01 0.10 1.00 10.00 -100 100 300 500 700 900 1100 1300 1500
  31. 31. PRESCOUTER What are the greatest challenges (technical, regulatory, or ethical) to broader applications of CRISPR within biomedical research, drug discovery, agricultural enhancements, or therapeutic applications? 6
  32. 32. PRESCOUTER What applications of CRISPR are you most excited to see in the next few years? What about the next 10–20 years? 7
  33. 33. PRESCOUTER 33 Future Direction of CRISPR in Pluripotent Stem Cells 1. Gene/Mutation Specific Drugs Patient-specific tissues or organs Drug screening Drug candidates for precision therapy FoldChangeofINS+ CellDeathRate #of INS+ cells 0.01 0.10 1.00 10.00 -100 100 300 500 700 900 1100 1300 1500 2. Isogenic iPSC-based Genome-Wide Association Study Genome-Wide Association Study Gene-Environment Interaction
  34. 34. THANK YOU STICK AROUND FOR OUR Q&A PRESCOUTER Your Personal Research Assistant

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