CRISPR Gene Therapeutics in Humans

Until recently, gene editing was extremely expensive and took several months, even years. However, with the discovery of CRISPR/Cas-9, costs have shrunk by 99% and tests can be run in days/weeks. Put very simply, the CRISPR/Cas9 technology is effectively a pair of microscissors that cuts DNA in a precise, directed manner. With CRISPR/Cas9, we can go into cells to modify DNA in a way that’s comparable to editing a document. Through this system, we have the potential to switch off the disease-causing mutated version of genes, delete fragments of DNA to treat conditions like Duchenne muscular dystrophy, and insert new genes to produce therapeutically relevant proteins.




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