Lecture-7Genome editing
CRISPR
Huseyin Tombuloglu, Phd
GBE423 Genomics & Proteomics
CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC
REPEATS (CRISPRS) AND CRISPR-ASSOCIATED (CAS) PROTEINS
CRISPR/CAS9 SYSTEM
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WHAT IS IT?
derived from a natural process found in bacteria to protect themselves from pathogens
targets genes for editing and regulating
comparable to Photoshop
Horizon Licenses Harvard University Gene-Editing Technology. (2013). Drug Discovery &
Development.4
Cas9 nuclease
sgRNA(single guide
RNA)
TO INITIATE GENE MODIFICATION,
Cas9 complex
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Gene of Interest
Protospacer Adjacent Motif (PAM)
Target Sequence
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Stop Codon
Non-Homologous End Joining (NHEJ) DNA repair pathway
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SUMMATION: CRISPR/CAS SYSTEM
sgRNA/Cas9
complex binds to
gene
Cas breaks gene
(double strand breaks)
Induced mutation in gene
sequence
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altered gene sequence dysfunctional CAD gene