Thursday 22 October 2020
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medicalxpress - 7 days ago

Rare congenital heart defect rescued by protease inhibition

A research team at the Greenwood Genetic Center (GGC) has successfully used small molecules to restore normal heart and valve development in an animal model for Mucolipidosis II (ML II), a rare genetic disorder. Progressive heart disease is commonly associated with ML II. The study is reported in this month s JCI Insight.


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