Article

Hematopoietic Stem Cell Gene Therapy Alleviates Disease Phenotypes in a Murine Model of Danon Disease

2022-08-19

Abstract excerpt

<title>Abstract</title> <p>Danon disease is a fatal X-linked recessive disease caused by a lack of expression of the lysosomal associated membrane protein type 2 (LAMP2), leading to severe vacuolar cardiomyopathy. Most patients with Danon progress to end-stage heart failure or death without advanced therapies. In this study, we investigated the therapeutic efficacy of systemic transplantation of <italic>ex vivo</...

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Literature Corpus work: 121d76d5-ee37-5684-b403-611b789e5852
DOI: 10.21203/rs.3.rs-1921280/v1

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