Tackling rare genetic disorders with patient-focused science
August 18, 2026
Shannon Knight, a brain and cognitive sciences PhD candidate and McGovern Institute researcher, focuses on developing a novel gene therapy.
Shannon Knight, a brain and cognitive sciences PhD candidate and McGovern Institute researcher, focuses on developing a novel gene therapy.
ANS-858 is being developed to reduce pathological craving associated with alcohol and other substance use disorders.
A study of worms finds a protein that helps shape the structure of the genome is critical for establishing the identity of some neurons.
Researchers from the Cancer Science Institute of Singapore (CSI Singapore) at the National University of Singapore (NUS) have uncovered a previously unknown mechanism that helps cancer cells evade detection by the body’s immune system.
Researchers at Tel Aviv University have developed a new method for diagnosing lung cancer: a simple, fast, low-cost blood test that does not require DNA sequencing.
A new study co-led by professor Valeriya Malysheva, group leader at the VIB-UAntwerp Center for Molecular Neurology, published in Nature Genetics, uncovers how the three-dimensional organization of DNA in rare immune cells can help explain genetic risk for Crohn’s disease and other autoimmune conditions.
A gene-editing tool designed to precisely rewrite the gene that causes Huntington’s disease reduced toxic protein fragments and symptoms associated with the disease in mice, researchers at the University of Illinois Urbana-Champaign report.
A single IV injection of a microRNA-based biologic developed by Guangping Gao, Ph.D., Robert H. Brown Jr., DPhil, MD, Jun Xie, Ph.D., and Zuoshang Xu, MD, Ph.D., suppressed production of the mutant SOD1 protein that causes amyotrophic lateral sclerosis (ALS), delayed disease onset by 60 days and extended lifespan by 100 days—more than tripling the average survival time—in mouse models of the disease.
Cancer immunotherapies have transformed treatment for many cancers, but pancreatic cancer remains especially difficult to treat. One major reason is that pancreatic tumors often create a “cold” tumor microenvironment, where the tumor prevents immune cells from mounting a strong attack.
Every encounter between a T cell and a potential target—especially when that target is a developing tumor—begins with a rapid series of molecular decisions. Within seconds, the immune cell must determine whether to launch an attack or stand down. T cells are so potent, so potentially devastating, that misreading the situation can cause serious tissue injury.