Canonical Wnt/β-catenin pathway activation can promote cardiomyocyte (CM) cell cycle activity, but translation is limited by off-target and functional liabilities associated with global pathway activation. We identified two small molecules, LRN9 and DF6, which potentiate Wnt/β-catenin signaling through a non-GSK3β mech...
Daniela T Asenjo, Lynn A. C. Devilée, Aaron H. Wasserman et al.· Journal of Molecular and Cel...· 0 citations
A dual-reporter and a high-throughput image-based pipeline are developed and a cardiomyocyte-selective pro-proliferative compound is identified as GDC-0339, uncovering cardiomyocyte-specific mechanisms governing the cell cycle that are potentially druggable.
It is demonstrated that adeno-associated virus-mediated delivery of full-length human ALPK3 prevents cardiomyopathy in neonatal mouse models and reverses established disease in adult mice, restoring cardiac structure, function and proteostasis, as well as contractile function in human cardiac organoids carrying ALPK3-...
James W. McNamara, Ellen B. Keen, Rebecca Sutton et al.· Nature Cardiovascular Resear...· 2 citations
Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common dominant muscular dystrophies and remains without an approved disease modifying therapy. Caused by the aberrant expression of the cytotoxic gene DUX4, FSHD is typically diagnosed in adulthood, however clinical onset in children (<18 years of age) i...
P. Houweling, Vanessa G. Crossman, L. Kiriaev et al.· bioRxiv· 0 citations
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