The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) Type II system is a bacterial immune system that has been modified for genome engineering. It is very powerful gene editing tool, with major potential in the medicinal field.

1. Presented by: Pragya Singh
CRISPR/CAS9

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The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) Type II system is
a bacterial immune system that has been modified for genome engineering. It is very
powerful gene editing tool, with major potential in the medicinal field.
This technology is quite cost-effective, very precise, efficient and flexible.
The two major components of CRISPR are:-
• A “guide” RNA (gRNA) which is a short synthetic RNA composed of a “scaffold”
sequence necessary for Cas9 binding and a user defined ∼20 nucleotide “spacer” or
“targeting” sequence also known as protospacer adjacent motif (PAM) which defines the
genomic target to be modified.
• A nonspecific CRISPR associated endonuclease (Cas9) which is basically RNA-guided
DNA endonuclease enzyme.
Introduction

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How CRISPR/Cas9 works
https://www.youtube.com/watch?v=2pp17E4E-O8
 The Cas9 enzyme cuts the DNA at the desired point
 The guide RNA (gRNA) will then latch on to the Cas9 enzyme
 Together they will search for the matching virus sequence or for the
mismatched sequence in the case of mutation
 Once they locate it Cas9 get to cutting and destroying it, thereby editing the
gene effectively.

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Application
 There are many potential applications of CRISPR/Cas9 technology. It can be
used in the medicinal field for the treatment of diseases like cystic fibrosis,
retinitis pigmentosa (RP) which leads to blindness, various cancers etc. etc. It
has also shown some real potential as a possible treatment for HIV in the
future.
https://www.youtube.com/watch?v=ZImVkl8QTW8

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