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PRESENTED BY: AHMED MADNI
REGISTRATION: SP14-BTY-011
 CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)
 Essential in adaptive immunity in selected bacteria and archaea,
enabling the organisms to respond to and eliminate invading genetic
material
 These repeats were initially discovered in the 1980s in E. coli
7 December 2015CRISPR/CAS9 2
 This technology is used to edit any genome sequence
 CRISPR has molecular scissor that can selectable disable or
change Gene sequence
 Used in Gene therapy treatment to cancer and Inherited disorder
7 December 2015CRISPR/CAS9 3
7 December 2015CRISPR/CAS9 4
7 December 2015CRISPR/CAS9 5
 Guide RNA
 Cas9 Endonuclease
7 December 2015CRISPR/CAS9 6
Non-homologous end joining
 Ligation method
Random mutation
Insertion/deletion
Gene knockout
Homologous direct repair
 Repair Templete
 Highly accurate method introduce
specific nucleotide
Gene knockout
Gene tagging
Specific mutation
Gene knockin
Promotor study
7 December 2015CRISPR/CAS9 7
7 December 2015CRISPR/CAS9 8
 Creating a
Double Strand
Break, while
minimizing off-
target effect
7 December 2015CRISPR/CAS9 9
• Transcriptional activator
• Transcriptional
repressor
• DNA labeling
7 December 2015CRISPR/CAS9 10
7 December 2015CRISPR/CAS9 11
 Simple
 Effective
 Use
Genetic therapeutics
Medicine
Cell and molecular research
7 December 2015CRISPR/CAS9 12
7 December 2015CRISPR/CAS9
13

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