Systemic AAV9.LAMP2B injection reverses metabolic and physiologic multiorgan dysfunction in a murine model of Danon disease
Ana Maria Manso, Sherin I. Hashem, Bradley Nelson, E.W. Gault, Angel Soto‐Hermida, Elizza E. Villarruel, Michela Brambatti, Julius Bogomolovas, Paul Bushway, Chao Chen, Pavan K. Battiprolu, Annahita Keravala, Jonathan D. Schwartz, Gaurav Y. Shah, Yusu Gu, Nancy D. Dalton, Kirk Hammond, Kirk L. Peterson, Paul Säftig, Eric Adler
Abstract
gene transfer improves metabolic and physiologic function in a DD murine model, suggesting that a similar therapeutic approach may be effective for treating patients with this highly morbid disease.
Topics & Concepts
DiseaseMedicineMultiorgan failureMultisystem diseaseInternal medicineCardiologyImmunologyCardiomyopathy and Myosin StudiesNeurogenetic and Muscular Disorders ResearchMuscle Physiology and Disorders