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Systemic AAV9.LAMP2B Injection Reverses Metabolic and Physiologic Multiorgan Dysfunction in a Murine Model of Danon Disease

Ana Maria Manso,Sherin 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

Science translational medicine（2020）

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