Systemic AAV9.LAMP2B injection reverses metabolic and physiologic multiorgan dysfunction in a murine model of Danon disease

Authors: Ana Maria Manso, Sherin I. Hashem, Bradley C. Nelson, Emily Gault, Angel Soto-Hermida, Elizza Villarruel, Michela Brambatti, Julius Bogomolovas, Paul J. Bushway, Chao Chen, Pavan Battiprolu, Annahita Keravala, Jonathan D. Schwartz, Gaurav Shah, Yusu Gu, Nancy D. Dalton, Kirk Hammond, Kirk Peterson, Paul Saftig, Eric D. Adler

Published: 2020-03-18

DOI: 10.1126/scitranslmed.aax1744

Source: Full article

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Abstract

AAV9.LAMP2B corrects metabolic and physiologic manifestations of a murine model of Danon disease.