July 16, 2018.
The Lupus Research Alliance is pleased and very encouraged to share a promising scientific breakthrough reported in the New York Times with real potential to revolutionize lupus treatment. Just reported in the prestigious journal Nature, scientists in Dr. Alexander Marson’s lab at the University of California, San Francisco have developed an innovative way to engineer genes of the body’s immune system to treat immunologic diseases like lupus, rheumatoid arthritis, cancer and HIV.
Building on decades of work, these scientists have used cutting-edge technology to quickly and efficiently insert beneficial genes at exact locations within human immune cells. Previously, this process was time-consuming, expensive and imprecise, introducing toxic viruses that could damage the cells.
“This new technology creates an opportunity to speed development and testing of therapeutic strategies in lupus by offering researchers new ways to modify particular genes that contribute to the disease,” comments Gerald Nepom, MD, PhD, Co-chair of the Lupus Research Alliance Scientific Advisory Board and Director of the Immune Tolerance Network (ITN), sponsored by the National Institute of Allergy and Infectious Diseases.
“The remarkable study describes improvements in genetic engineering that allow the modification of multiple genes simultaneously in specific immune system cells,” said Ward Wakeland, PhD, Edwin L. Cox Distinguished Chair in Immunology and Genetics at UT Southwestern Medical Center. “As a result, proposing to change several defective genes simultaneously in a single therapeutic treatment is now feasible. By coupling this technology with a detailed understanding of the specific genetic variations that cause lupus in individual patients, it should be technically possible to design patient-specific therapies that suppress autoimmune disease without completely impairing the immune system.”
Richard DeScherer, Co-chair of the Lupus Research Alliance Board of Directors expressed the organization’s excitement at these new findings. “This is exactly the level of modern immunology and powerful new technology that we seek. This is the revolutionary science that can address the complexities of the disease and advance understanding of lupus at the molecular level, to carve out the path to the most effective new treatments and transform the lives of people with lupus.”
Lupus is a chronic, complex autoimmune disease that affects millions of people worldwide. More than 90% of people with lupus are women; lupus most often strikes during the childbearing years of 15-45. African Americans, Latin Americans, Asians and Native Americans are two to three times at greater risk than Caucasians. In lupus, the immune system, which is designed to protect against infection, creates antibodies that can attack any part of the body including the kidneys, brain, heart, lungs, blood, skin, and joints.
About the Lupus Research Alliance
The Lupus Research Alliance aims to transform treatment while advancing toward a cure by funding the most innovative lupus research in the world. The organization’s stringent peer review grant process fosters diverse scientific talent who are driving discovery toward better diagnostics, improved treatments and ultimately a cure for lupus. Because the Lupus Research Alliance’s Board of Directors fund all administrative and fundraising costs, 100% of all donations goes to support lupus research programs.