Genome Editing May Stop Heart Disease for Good

the MPR take:

Genomic editing of the PCSK9 gene in humans may be the key to stopping cardiovascular disease, says a study published in the journal Circulation. By disrupting the PCSK9 gene in mice, researchers were able to reduce their blood cholesterol levels; mutation of the gene led to increased hepatic LDL receptor levels  and decreased plasma cholesterol levels. Humans with a naturally occurring PCSK9 mutation have been shown to have less LDL cholesterol in the blood, so researchers are hoping that the results achieved with mice will have therapeutic potential in preventing heart disease in humans. While drug companies are also working on targeting the PCSK9 gene with pharmacological agents, genomic editing would permanently protect a patient from high LDL levels.

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Genome Editing May Stop Heart Disease for Good

Doctors, and especially doctors who do research, don't like to use the words cure or eradicate. Taking advantage of advances in genetic engineering, a team lead by Musunuru, who holds positions at Harvard University's Department of Stem Cell and Regenerative Biology and Brigham and Women's Hospital, have edited the genomes of mice and successfully protected them from heart disease.