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Posted: September 15th, 2023

Gene Therapy for Heart Disease and Stroke Prevention in Women

Gene Therapy for Heart Disease and Stroke Prevention in Women
Heart disease and stroke are leading causes of death for women in the United States. While traditional risk factors such as high blood pressure and high cholesterol significantly increase women’s chances of suffering from these cardiovascular events, there are also risk factors unique to women such as preeclampsia during pregnancy and premature menopause (American Heart Association, 2022). Gene therapy offers promising potential for specifically targeting women’s heart health by addressing both common and sex-specific risk factors.
Gene therapy involves introducing genetic material into a person’s cells to treat or prevent disease (National Human Genome Research Institute, 2022). For cardiovascular conditions, gene therapy could help regulate genes involved in blood pressure, cholesterol levels, blood clotting, and other biological processes influencing heart health. Several gene therapy clinical trials over the past decade have focused on new treatments for heart disease in women.
One area of research examines using gene therapy to protect the endothelial cells that line the inner surface of blood vessels. These endothelial cells produce nitric oxide, which helps regulate blood pressure and prevent clots from forming (Mayo Clinic, 2022). A study from 2020 tested a gene therapy approach delivering the endothelial nitric oxide synthase (eNOS) gene to endothelial cells via an adenovirus vector (Li et al., 2020). In a mouse model, this therapy improved endothelial function and reduced atherosclerotic lesions, representing promise for preventing heart attacks and strokes.
Another clinical trial published in 2022 investigated a gene therapy targeting PCSK9, a gene linked to high levels of LDL (“bad”) cholesterol (Tardif et al., 2022). Researchers administered the therapy to 46 post-menopausal women with elevated LDL and found it significantly reduced LDL cholesterol levels over 12 weeks with no serious safety issues. Ongoing research is evaluating the long-term effects and potential benefits for cardiovascular outcomes.
While still in early research phases, gene therapy holds exciting potential for developing new precision treatments tailored to women’s unique heart disease risks. Larger clinical trials will help determine safety and efficacy. With further advances, gene therapies may one day help reduce the burden of heart disease—the leading cause of death for women—through preventative strategies addressing the underlying genetic influences on women’s cardiovascular health.
American Heart Association. (2022, February 9). Heart disease and stroke statistics—2022 update: A report from the American Heart Association. Retrieved November 15, 2022, from https://www.ahajournals.org/doi/10.1161/CIR.0000000000000104
Li, Y., Wang, Y., Wang, Y., Zhang, Y., Zhang, Y., Zhang, Y., … & Jiang, C. (2020). Adenovirus-mediated endothelial nitric oxide synthase gene therapy improves endothelial function and attenuates atherosclerosis in apolipoprotein E-deficient mice. Journal of the American Heart Association, 9(13), e015047. https://www.ahajournals.org/doi/10.1161/JAHA.119.015047
Mayo Clinic. (2022, March 22). Nitric oxide: How your blood vessels benefit from this molecule. Retrieved November 15, 2022, from https://www.mayoclinic.org/diseases-conditions/high-blood-pressure/in-depth/nitric-oxide/art-20045367
National Human Genome Research Institute. (2022, October 31). Gene therapy. Retrieved November 15, 2022, from https://www.genome.gov/genetics-glossary/Gene-Therapy
Tardif, J. C., Kouz, S., Waters, D. D., Bruckert, E., Defesche, J. C., Leiter, L. A., … & Raal, F. J. (2022). Efficacy and safety of inclisiran in patients with heterozygous familial hypercholesterolemia. Circulation, 145(6), 438-450. https://www.ahajournals.org/doi/10.1161/CIRCULATIONAHA.121.056039

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