Mount Sinai researchers develop new gene therapy for heart failure & related general gene therapy Web sites and resources
Researchers at Mount Sinai School of Medicine have found in a Phase II trial that a gene therapy developed at Mount Sinai stabilized or improved cardiac function in people with severe heart failure. Patients receiving a high dose of the therapy, called SERCA2a, experienced substantial clinical benefit and significantly reduced cardiovascular hospitalizations, addressing a critical unmet need in this population. The data are published online in the June 27 issue of the American Heart Association journal Circulation.
SERCA2a is delivered via an adeno-associated virus vector—an inactive virus that acts as a medication transporter—into cardiac cells. The therapy stimulates production of an enzyme within these cells that enables the heart to pump more effectively in people with advanced heart failure. After one year, patients who were administered a high dose SERCA2a demonstrated improvement or stabilization. Gene therapy with SERCA2a was also found to be safe in this sick patient population, with no increases in adverse events, disease-related events, laboratory abnormalities, or arrhythmias compared to placebo….
A sampling of general gene therapy resources
- Genes and gene therapy (MedlinePlus) has links to overviews, latest news, specific conditions, organizations, directories, and more
- Genetics home reference (US National Institutes of Health) with links to information on over 600 conditions/diseases, information on over 600 genes, a handbook with basic gene related information, a glossary, and links to additional resources
- Genetics education center (University of Kansas) with links to education resources, Human Genome Project materials, activities, and more
- Learn Gentics (University of Utah) includes basic information and research related concepts. Extensive animations and videos.
- New gene therapy fixes mistakes (sciencenews.org)
- Genome Editing Improves Blood Clotting in Mice with Hemophilia B (nextbigfuture.com)
- Advances in delivery of therapeutic genes to treat brain tumors (medicalxpress.com)
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