Scientists from the United States have presented a new method of gene editing for the treatment of rare diseases. It involves the introduction of nanoparticles into the blood.
The researchers explained that editing the CRISPR genes is already helping to fight diseases that were previously considered incurable. However, until now, methods have required injection directly into the affected cells. According to NPR, researchers have now published results showing that CRISPR-Cas9 can be injected into the bloodstream to make changes, which opens up the possibility of using gene editing to treat many common diseases.
Experimental treatment is aimed at combating a 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.
The trial involved only six people, and the research team has yet to conduct long-term studies to test for 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 already fear the potential abuse 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.