Isoprenylcysteine Carboxylmethyltransferase-Based Therapy for Hutchinson–Gilford Progeria Syndrome
Beatriz Marcos‐Ramiro, Ana Gil-Ordóñez, Nagore I. Marín‐Ramos, Francisco J. Ortega-Nogales, Moisés Balabasquer, Pilar Gonzalo, Nora Khiar‐Fernández, Loïc Rolas, Anna Barkaway, Sussan Nourshargh, Vicente Andrés, Mar Martín‐Fontecha, María L. López-Rodrı́guez, Silvia Ortega‐Gutiérrez
Abstract
progeroid mouse model, UCM-13207 showed an excellent in vivo efficacy by increasing body weight, enhancing grip strength, extending lifespan by 20%, and decreasing tissue senescence in multiple organs. Furthermore, UCM-13207 treatment led to an improvement of key cardiovascular hallmarks such as reduced progerin levels in aortic and endocardial tissue and increased number of vascular smooth muscle cells (VSMCs). The beneficial effects go well beyond the effects induced by other therapeutic strategies previously reported in the field, thus supporting the use of UCM-13207 as a new treatment for progeria.