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For the first time, bioscientists have cured a genetic defect with an injection.
The defective gene spawns a rare illness called transthyretin amyloidosis, in which the liver makes a malformed protein that accumulates in the heart, eventually causing the heart to stop.
In its first test, the one-time injection halted almost all production of the guilty protein in three of six volunteers.
The treatment joins CRISPR, the Nobel-winning gene-editing technology, with the expanding use of messenger RNA (mRNA), the foundation of several successful COVID vaccines.
The volunteers were injected with fat particles containing two messenger RNAs. One told the body to make Cas protein, which cuts DNA. The other RNA steered the first one to the location on the DNA helix where the gene for the defective protein resides.
After Cas cut out the defective gene, the body healed the broken DNA strand but without the faulty gene included. Result: no more malformed protein.
After 28 days, three of the volunteers showed 81 percent to 96 percent less of the unwanted protein.
The test is “a critical first step in being able to inactivate, repair, or replace any gene that causes disease, anywhere in the body,” Jennifer Doudna, CRISPR’s co-inventor, said in a statement praising the result.
TRENDPOST: Scientists have been using CRISPR to edit genes for several years but have lacked a means to get the body to edit its own genes.
For that reason, the impact of this successful trial is, for genetic medicine, equivalent to Steve Wozniak’s invention of the personal computer: it will create not only an entire new industry, but also has the power to reshape the world we live in.
Specifically, physicians now have the expectation to, one day, be able to correct genetic illnesses with a quick and easy treatment. Those genetic corrections should be inheritable, meaning that we can now realistically envision inherited conditions ranging from male pattern baldness to brain-wasting Huntington’s Disease being engineered out of existence.