Using CRISPR-Cas9, researchers say they can reverse damage after a heart attack in mice, showing the exciting potential of broader uses for this decade-old gene editing technology.
The challenge: After a heart attack, damage to the heart grows steadily worse, driven by overactivation of a gene called CaMKIIδ. The mechanism is two methionine amino acids within the CaMKIIδ protein, according to scientists at the University of Texas Southwestern Medical Center.
The proposed solution: Using a viral, RNA-based delivery system that directly targets the heart, combined with a base editor to alter the CaMKIIδ gene, the UT team converted the methionine into a different, harmless amino acid.
The result: Mice receiving the treatment after a heart attack recovered almost completely, and CaMKIIδ was unchanged in the rest of their bodies. Meanwhile, the condition of untreated post-heart attack mice deteriorated steadily.
Why it matters: While there has been much investigation of CRISPR Cas9 to treat rare genetic diseases, treating a non-genetic condition—especially one as common as a heart attack—is an exciting new direction, says Dr. Rhonda Bassel-Duby, a co-leader of the study.
Expect further progress: Scientists have been anticipating this expansion of the uses of CRISPR to encompass a range of diseases and even prevention, according to an interview with Bio.News.
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