1. Lecture-7 Genome editing CRISPR
Huseyin Tombuloglu, Phd
GBE423 Genomics & Proteomics

3. Clustered regularly interspaced short palindromic repeats (CRISPRs) and CRISPR-associated (Cas) proteins
CRISPR/Cas9 System

4. 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.

5. To initiate gene modification,
sgRNA
(single guide RNA)
Cas9 complex
Cas9 nuclease

6. Protospacer Adjacent Motif (PAM)
Target Sequence
Gene of Interest

7. Non-Homologous End Joining (NHEJ) DNA repair pathway
Stop Codon

8. Summation: crispr/Cas system
sgRNA/Cas9 complex binds to gene
Cas breaks gene (double strand breaks)
Induced mutation in gene sequence
altered gene sequence  dysfunctional CAD gene