亨廷顿氏病,动物模型,ATM信号 z-bo 2020-09-17 363

　　ATM research has shown that limiting the effect of ATM protein can protect brain cells from death in animal models of Huntington's disease. Huntington's disease is an inherited neurodegenerative disease in which fragments of the mutant protein Huntingtin can block and damage nerve cells in the brain, thereby impairing motor and cognitive abilities.

　　The cause of the disease is still poorly understood, which limits the development of effective treatments for the disease. Xiao-HongLu and colleagues studied the ATM signaling pathway that plays a central role in repairing DNA damage. When the damage is irreversible, ATM can cause cell suicide. In fact, researchers have found abnormally elevated ATM signaling in the brain tissue of mice and patients with Huntington's disease.

　　Then, we tested the effect of reducing ATM in many cells and animal models, including mouse, flies, rat cells and pluripotent stem cells derived from Huntington's disease patients. Reducing ATM can completely protect brain cells from the toxicity of mutant huntingtin fragments, reduce cell death and partially prevent disease progression (for example, these mice even show improved athletic performance and behavior). ). These results provide good reasons for exploring the use of ATM inhibitors as possible treatments for Huntington's disease; ATM inhibitors have been developed in the past to treat cancer.