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ALL OF IT LEADING TO THE DAYS OF NOAH? CRISPR Gene Therapy Breakthrough Could Be A ‘One And Done’ Injection

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The therapy is made up of three parts. A tiny bubble of fat, called a lipid nanoparticle, carries a payload of CRISPR machinery: a strand of guide RNA and a sequence of mRNA coding for the Cas9 protein. Billions of these CRISPR-carrying nanoparticles are infused into the bloodstream, making their way to the source of the dysfunctional protein. The mRNA instructs the cells to produce the Cas9 protein (CRISPR’s genetic ‘scissors’) which then links up with the guide RNA, seeks out the target gene, and snips it. The cell repairs the DNA at the site of the break, but imperfectly, switching the gene off and shutting down production of the problematic protein. Interim trial results, reported in the New England Journal of Medicine last weekend, were very encouraging.  The CRISPR approach, if successful, would be a one-time treatment. That is, by targeting the genes themselves, the protein is permanently silenced… (READ MORE)

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