Gene therapy of hereditary arrhythmia
CRISPR prevents sudden death from arrhythmia
Svetlana Maslova, Hi-tech+
Scientists with the help of gene editing cured mice of hereditary cardiac arrhythmia. Targeted editing is the future of gene therapy, say enthusiastic Baylor University specialists.
Cardiovascular diseases cause the death of about 3 million people worldwide every year, and hereditary arrhythmia is responsible for a significant proportion of deaths. Researchers from Baylor University (USA) have used adeno-associated viruses to deliver CRISPR/Cas9 genome editing tools directly to the heart.
The first successful experiments were conducted on mice: scientists managed to cure all rodents with the disease-associated mutation R176Q with a single injection and selective destruction of the mutant gene, according to the university's website.
The scientists divided the mice into two groups, where one was injected into the heart ten days after birth, and the other received a placebo.
As a result, no arrhythmia was observed in rodents from the first group for six weeks after treatment, and in the placebo group, 71% of the disease manifested itself.
In addition, editing a defective copy of the gene significantly reduced the presence of non-functional RYR2 proteins, and the only healthy copy of the gene synthesized a normal protein in sufficient quantity to maintain proper heart function. No side effects were observed.
"We are glad that we were able to selectively destroy the disease-causing gene without affecting healthy copies in the genome. Targeted editing is the future of gene therapy, and cardiovascular diseases seem to us an ideal "platform" to start," concludes researcher Xander Verens.
Now the team is testing a similar approach in stem cells of patients with a similar diagnosis to confirm the safety and effectiveness of the technique for humans.
CRISPR technology is able to correct a number of important mutations, but the approach is still limited to the area of the genome that this method can correct. Scientists have developed an auxiliary tool that increases the possibilities of gene editing almost twice.
Article by Pan et al. In Vivo Ryr2 Editing Corrects Catecholaminergic Polymorphic Ventricular Tachycardia published in the journal Circulation Research.
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