The advancement of CRISPR gene editing could treat many more diseases
CRISPR genetic editing already promises to fight disease which were once thought unassailable, but techniques so far have required injecting the tools directly into the affected cells. It is not very practical for some conditions. However, there was only one adventure. NPR report that researchers have published results showing that you can inject CRISPR-Cas9 into the bloodstream to make changes, opening the door to the use of genetic editing to treat many common diseases.
Experimental treatment treated a rare genetic disease, transthyretin amyloidosis. The scientists injected volunteers with nanoparticles loaded with CRISPR that were absorbed by the patients ’livers, editing a gene in the organ to disable the production of a harmful protein. Levels of that protein precipitate within weeks of injection, saving patients from a disease that can quickly destroy nerves and other tissues in their body.
The test involved only six people, and the research team should always conduct long-term studies to check for any adverse effects. If this method proves feasible on a large scale, however, it could be used to treat diseases where existing CRISPR techniques are not practical, from Alzheimer’s to heart disease.
There are some ethical considerations. Some are already paying attention to the potential for abuse of CRISPR for ‘creatures of conception’ and other less altruistic purposes. Blood circulation injections will make it so much easier to make dubious changes. However, if used correctly, this new CRISPR method could avoid (or prevent) the suffering that was once considered inevitable.
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