CRISPR Gene Editing promises to fight incurable diseases but techniques have required injecting the tools directly into affected cells. NPR reports that researchers have published results showing that you can inject CRISPR-Cas9 into the bloodstream to make edits, opening the door to the use of gene editing for treating many common diseases.
The experimental treatment fought a rare genetic disease, transthyretin amyloidosis. Scientists injected volunteers with CRISPR-loaded nanoparticles 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 plunged within weeks of the injection, saving patients from an illness that can rapidly destroy nerves and other tissues in their bodies.
The test involved just six people, and the research team still has to conduct long-term studies to check for possible negative effects. If this method proves achievable on a large scale, it could be used to treat illnesses where existing CRISPR Gene Editing techniques aren’t practical, varying from Alzheimer’s to heart disease.There are some ethical considerations. Some are already wary about the potential for abusing CRISPR Gene Editing for designer babies and other less-than-altruistic purposes. Bloodstream injections would make it that much easier to perform dubious edits. This new CRISPR method could avoid the suffering that was once considered inevitable.
The early trial data from Intellia Therapeutics, co-founded by Nobel Prize winner Jennifer Doudna, marked a breakthrough for Crispr-based treatments, showing scientists had overcome challenges that had previously restricted the technology’s use to editing cells outside the body or in the eye. Shares in Intellia have risen 233 per cent since it went public in 2016.
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