New CRISPR Injection Method Will Help Treat Rare Diseases

The researchers explained that CRISPR gene editing is already helping to fight diseases that were previously

were considered incurable.However, until now, methods have required injection directly into affected cells. As NPR reports, researchers have now published results showing that it is possible to inject CRISPR-Cas9 into the blood to make changes, opening up the possibility of using gene editing to treat many common diseases.

Experimental treatment is aimed at combatinga rare genetic disease - transthyretin amyloidosis. Scientists injected volunteers with nanoparticles with CRISPR, which were absorbed by the liver of patients, editing a gene in that organ to turn off the production of harmful protein. This protein dropped a few weeks after the injection, saving patients from disease that can quickly destroy nerves and other tissues in the body.

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Only six people participated in the test, andthe research team has yet to conduct long-term studies to verify possible negative effects. However, if this method proves to be viable on a large scale, it could be used to treat conditions for which existing CRISPR methods are inapplicable, from Alzheimer's to heart disease.

However, some are already wary of the potentialabuse of CRISPR, and injections will make it much easier to make questionable changes. However, when used correctly, the new CRISPR technique can prevent diseases that are suffering that were previously thought to be inevitable.

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