CRISPR
Gene Editing is just the
Beginning
Presented By :
Akash Arora (1410105001)

AsWikipedia says :
Genome editing is a technique where DNA is
inserted,replaced or removed from a genome using
artificially engineered nucleases.
The nucleases create specific double-strand breaks (DSBs)
at desired locations in the genome and harness the cell’s
endogenous mechanisms to repair the induced break by
natural processes of homologous recombination (HR)
and non-homologous end-joining (NHEJ).
 Genome Editing

 Approaches of Gene editing?

 Story Behind CRISPR
198
7
• Researchers find CRISPR sequences in Escherichia
coli, but do not characterize their function.
200
0
• CRISPR sequence are found to be common in other
microbes.
200
2
• Coined CRISPR name, defined signature Cas genes
200
7
• First experimental evidence for CRISPR adaptive
immunity
201
3
• First demonstration of Cas9 genome engineering in
eukaryotic cell

 CRISPR: Clustered Regularly Interspaced Short
Palindromic Repeats
 First described in E. coli and determined to be part of the bacterial
innate immune system versus bacteriophages
 Consists of short segments of DNA that are palindromes interspaced
with spacer DNA
 The spacer DNA is identical in sequence to viral (bacteriophage) DNA
 There are additionalCRISPR associated proteins:
cas proteins that are typically helicases or nucleases

• CRISPR/Cas system is a prokaryotic immune system that confers
resistance to foreign genetic elements such as phages.
• Much easier to design compared to its predecessors zinc finger
nucleases andTALENs.
• CRISPR loci range from 24 to 48 bp.
• CRISPR-associated(cas) genes are attached to the
repeater-spacer array
• Spacers are fragments of DNA gathered
from the viruses that had tried to attack
 Some More Important Points

The CRISPR immune system works to protect
bacteria from repeated viral attack via three basic
steps:
 Adaptation
 Production of cr RNA
 Targeting
 How it works

 Structure of cas9 protein

 Structure of crRNA

 Clinical Human Applications of CRISPR
• Genetic diseases
Remove or add the sequence that is causing the
disease
• Transplantation
Gene editing of mismatched human or even non-human
mammals as potential
organ donors
Editing will reduce risk of immune responses and rejection
when using
mismatched organs.

• Drug development – optimize biotech
manufacture
• Disease models
• Ecological vector control – mosquito
sterilization
• Biofuels
 Some more Important Application

• CRISPR system are advantageous for industrial
processes that utilize bacterial cultures.
• CRISPR-based immunity can be employed to
make these cultures more resistant to viral
attack, which would otherwise impede
productivity.

Companies using CRISPR

 Software and databases

 No. of CRISPR paper published year wise

 Rise in Funds

 Key Players

• CRISPR technology has emerged as a powerful and
universal technology for genome engineering with wide-
ranging innovative implications across biology and
medicine.
• This technology has proved its potential by being user
friendly and has shown its practicality in ensuring health as
well as food security of the future.
• The tool itself do not pose a threat and we hope that the
CRISPR technology will live up to its promise by being used
Impact of CRISPER