Breakthrough CRISPR Gene Therapy Could Be a ‘One and Done’ Injection

A growing number of clinical trials are beginning to test gene therapies in humans.. Although cutting-edge, the therapies can be costly and intense.. doctors remove cells from the body, edit them in a dish, and then infuse them back into the patient. So researchers are looking for a general delivery method, like any other medication. Now, a clinical trial from University College London (UCL) has taken a step in that direction.

Patients received a single infusion of a CRISPR-based therapy into their bloodstream. Blood carried the therapy to the liver, where it switched off the mutated gene and curtailed production of the errant protein..

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.. 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.

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Just like the vaxxine then. This will replace all medical treatment in the future I suspect.

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