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