Key result
Systemic AAV9.LAMP2B gene transfer improves metabolic and physiologic multiorgan dysfunction in a murine model of Danon disease.
Why the study?
Does systemic AAV9.LAMP2B injection improve metabolic and physiologic function in a murine model of Danon disease?
Does systemic AAV9.LAMP2B injection improve metabolic and physiologic function in a murine model of Danon disease?
Systemic AAV9.LAMP2B gene transfer improves metabolic and physiologic function in a murine model of Danon disease, suggesting a potential therapeutic approach for patients.
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Hypothesis-generating for AAV9.LAMP2B gene therapy in Danon disease; human trials needed before clinical consideration.
Manso et al. (2020) studied this question. Systemic AAV9.LAMP2B gene transfer improves metabolic and physiologic multiorgan dysfunction in a murine model of Danon disease.
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