SlideShare a Scribd company logo
1
2
Jennifer A.
Doudna
Emmanuelle
Charpentier
The Royal Swedish Academy of Sciences in Stockholm awarded the two
scientists “for the development of a method for genome editing.”.
3
The new frontier of
Genome
Engineering
with
CRISPR-Cas9
Jennifer A. Doudna and Emmanuelle Charpentier
4
28 NOVEMBER 2014
Year of publicationYear of publication
5
VOL 346 ISSUE 6213
Science (print ISSN 0036-8075; online ISSN 1095-9203)
is published weekly, except the last week in December,
by the American Association for the Advancement of Science
(AAAS), 1200 New York Avenue NW, Washington, DC
20005
6
CRISPR/Cas9 is a gene editing tool that can manipulate gene
expression in plants, humans and animals. CRISPR, or
Clustered Regularly Interspaced Short Palindromic Repeats,
are short sections of bacterial DNA containing repetitive base
sequences
The simple two-component CRISPRCas9 system, using
Watson-Crick base pairing by a guide RNA to identify target DNA
sequences, is a versatile technology that has already stimulated
innovative applications in biology.
Summary
7
The simple two-component CRISPRCas9 system, using
Watson-Crick base pairing by a guide RNA to identify target DNA sequences, is a
versatile technology that has already stimulated innovative applications in biology.
8
Description
• sgRNA (light green)
• tracrRNA (dark green)
• PAM (red)
CRISPR-Cas9 development
9
The CRISPR-Cas9 technology originates from type II CRISPR-Cas systems, which
provide bacteria with adaptive immunity to viruses and plasmids.
10
Understanding the CRISPR-Cas9 system at the biochemical and structural level
allows the engineering of tailored Cas9 variants with smaller size and increased
specificity.
The CRISPR/Cas9 system then can store this viral data so that it will be able to
recognize and eliminate future viral threats.
Specific methods for delivering Cas9 and its guide RNA to cells and
tissues should benefit the field of human gene therapy.
The versatility of the CRISPR/Cas9 system and its ability to locate and
alter specific genes can yield advancements in drug discovery,
basic medical research, agriculture and even the possibility of preventing
genetic diseases, heart disease, and blood conditions in humans.
11
12
13
Timeline of CRISPR-Cas9 and genome engineering
research fields
14
15
S. Scherer, R. W. Davis, Replacement of chromosome
segments with altered DNA sequences constructed in vitro.
Proc. Natl. Acad. Sci. U.S.A. 76, 4951–4955 (1979).
doi: 10.1073/pnas.76.10.4951; pmid: 388424
Reference
16
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for
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CRISPR-Cas9: The new frontier of Genome Engineering

  • 1. 1
  • 2. 2 Jennifer A. Doudna Emmanuelle Charpentier The Royal Swedish Academy of Sciences in Stockholm awarded the two scientists “for the development of a method for genome editing.”.
  • 3. 3 The new frontier of Genome Engineering with CRISPR-Cas9 Jennifer A. Doudna and Emmanuelle Charpentier
  • 4. 4 28 NOVEMBER 2014 Year of publicationYear of publication
  • 5. 5 VOL 346 ISSUE 6213 Science (print ISSN 0036-8075; online ISSN 1095-9203) is published weekly, except the last week in December, by the American Association for the Advancement of Science (AAAS), 1200 New York Avenue NW, Washington, DC 20005
  • 6. 6 CRISPR/Cas9 is a gene editing tool that can manipulate gene expression in plants, humans and animals. CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, are short sections of bacterial DNA containing repetitive base sequences The simple two-component CRISPRCas9 system, using Watson-Crick base pairing by a guide RNA to identify target DNA sequences, is a versatile technology that has already stimulated innovative applications in biology. Summary
  • 7. 7 The simple two-component CRISPRCas9 system, using Watson-Crick base pairing by a guide RNA to identify target DNA sequences, is a versatile technology that has already stimulated innovative applications in biology.
  • 8. 8 Description • sgRNA (light green) • tracrRNA (dark green) • PAM (red) CRISPR-Cas9 development
  • 9. 9 The CRISPR-Cas9 technology originates from type II CRISPR-Cas systems, which provide bacteria with adaptive immunity to viruses and plasmids.
  • 10. 10 Understanding the CRISPR-Cas9 system at the biochemical and structural level allows the engineering of tailored Cas9 variants with smaller size and increased specificity. The CRISPR/Cas9 system then can store this viral data so that it will be able to recognize and eliminate future viral threats. Specific methods for delivering Cas9 and its guide RNA to cells and tissues should benefit the field of human gene therapy. The versatility of the CRISPR/Cas9 system and its ability to locate and alter specific genes can yield advancements in drug discovery, basic medical research, agriculture and even the possibility of preventing genetic diseases, heart disease, and blood conditions in humans.
  • 11. 11
  • 12. 12
  • 13. 13 Timeline of CRISPR-Cas9 and genome engineering research fields
  • 14. 14
  • 15. 15 S. Scherer, R. W. Davis, Replacement of chromosome segments with altered DNA sequences constructed in vitro. Proc. Natl. Acad. Sci. U.S.A. 76, 4951–4955 (1979). doi: 10.1073/pnas.76.10.4951; pmid: 388424 Reference