Breaking the Skin Barrier: Drugs Topically Deliver Gene Therapy Via Commercial Moisturizers for Skin Disease Treatment
Drugs are first to topically deliver gene regulation via commercial moisturizers for skin disease
Hand Cream – A new class of nanoparticles inside skin lotion could penetrate the skin for gene therapy.
Getting under your skin” takes on a brave new meaning thanks to Northwestern University research that could transform gene regulation.
A team led by a physician-scientist and a chemist — from the fields of dermatology and nanotechnology — is the first to demonstrate the use of commercial moisturizers to deliver gene regulation technology that has great potential for life-saving therapies for skin cancers.
The topical delivery of gene regulation technology to cells deep in the skin is extremely difficult because of the formidable defenses skin provides for the body. The Northwestern approach takes advantage of drugs consisting of novel spherical arrangements of nucleic acids. These structures, each about 1,000 times smaller than the diameter of a human hair, have the unique ability to recruit and bind to natural proteins that allow them to traverse the skin and enter cells.
Applied directly to the skin, the drug penetrates all of the skin’s layers and can selectively target disease-causing genes while sparing normal genes. Once in cells, the drug simply flips the switch of the troublesome genes to “off.”
A detailed study of a method that could dramatically redefine the field of gene regulation will be published online during the week of July 2 by the Proceedings of the National Academy of Sciences (PNAS)….
- Breaking the skin barrier: Drugs topically deliver gene therapy via commercial moisturizers for skin disease treatment (sciencedaily.com)
- Breaking the skin barrier (eurekalert.org)
- A Topically Applied Skin Lotion That Modifies Your Genes (sott.net)
- Gene healing in a lotion? Researchers say they’re close (vitals.msnbc.msn.com)
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)