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Human Genetic Diseases At Salk University Essay

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The CRISPR-Cas9 research and recent advances in animal models are showing promise to treat lots of human genetic diseases in the future. In a mainstream science article published by IFLScience!, they highlight research being done at Salk University that is working on using CRISPR-Cas9 and HITI technology to target non-dividing cells of the eye to help improve vision in rats with retinitis pigmentosa.3 The article briefly mentions what the CRISPR-Cas9 system is, stating that CRISPR is a method that snips strands of DNA and replaces those with new genetic material.3 Other than the effects of CRISPR research, and what it could mean for the future of genetic diseases, IFLScience! fails to mention the complexity of how the complex works, the engineered specificity of it, and the problems with current delivery.

The CRISPR-Cas9 complex is derived from the immune system of bacterial cells and also contains repurposed exogenous RNA’s responsible for editing the human genome.1 To edit a gene within the genome, researchers add a CRISPR RNA (crRNA), which is complementary to the DNA code of interest and is responsible for binding. The crRNA is engineered to be extremely specific to the code of interest. A trans-activating CRISPR RNA (tracrRNA) is also necessary for guiding all the pieces of the complex together to carry out the function of editing. The repurposed CRISPR-Cas9 complex contains two nucleases, RuvC and HNH, which perform noncomplementary strand cleavage and complementary

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