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The background of CRISPR CRISPR is an ancient bacterial adaptive immune system, evolved to take a record of the DNA or RNA of incoming phages, so it can destroy them later. It makes use of programmable scissors, that can be easily targeted to any sequence -- allowing them to cut the invading virus. DNA DNA HOST DNA(GENOME) CAS 9 CAS GENES LEADER REPEAT REPEAT REPEAT SPACER SPACER
1 Spacer Acquisition Viral DNA fragments are incorporated as spacers in the CRISPR locus 2 Target Degradation These components encode a program of programmable targeting against nucleic acid targets 3 Cas9 production and crRNA expression Once the target sequence is recognized, the endonuclease activity of the Cas protein is activated, leading to target cleavage. Cas9 GUIDE RNA crRNA Target Cleavage
Therapeutic Applications of CRISPR CRISPR-Cas9 has been leveraged for programmable gene editing, enabling applications ranging from foundation studies investigating gene function to permanently correcting heritable disease. The pace of Cas9 innovation is transforming the face of science and making it easier to generate preclinical models of disease. It has also revolutionized multiple industries including gene therapy, plant and agricultural engineering, and even traditional pharma.
1 Gene Therapy A cell is transfected with an enzyme complex: Guide RNA, a healthy DNA template, and a CRISPR-Cas9 are delivered to a cell 2 Targeting & Cleavage The specifically designed guide RNA finds the target DNA strand, which Cas9 cuts 3 Repair The DNA cut is corrected using the healthy DNA copy that is co-delivered as a repair template, restoring the mutated DNA back to normal
cell chromosome GUIDE RNA Cas9 Protein Cas9 CUTS THE TARGET DNA STRAND GENOMIC DNA GENOMIC DNA Repaired DNA
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