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a) A diagram and brief introduction to the disease and how the mutation gives rise to it

01 / 10 / 2021 Medical

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CRISPR-Cas9 gene editing by NHEJ is generally more efficient than HDR, but correction of point mutations in an open reading frame is only really feasible by HDR. However, a recent paper (He 2016) describes a new method for inserting DNA segments into the genome at precise regions by NHEJ. 
Question

In a genetic disease of your choice (except CF), outline a protocol to correct a known mutation that causes that disease.Structure your answer as follows: 
a)    A diagram and brief introduction to the disease and how the mutation gives rise to it 



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