Article
Fasudil alleviates the vascular endothelial dysfunction and several phenotypes of Fabry disease.
Molecular therapy : the journal of the American Society of Gene Therapy - 5 Apr 2023
Choi Jong Bin, Seol Dong-Won, Do Hyo-Sang, Yang Hee-Young, Kim Taek-Min, Byun Youkyeong Gloria, Park Jae-Min, Choi Jinhyuk, Hong Seon Pyo, Chung Won-Suk, Suh Jae Myoung, Koh Gou Young, Lee Beom Hee, Wee Gabbine, Han Yong-Mahn
Abstract excerpt
Fabry disease (FD), a lysosomal storage disorder, is caused by defective α-galactosidase (GLA) activity, which results in the accumulation of globotriaosylceramide (Gb3) in endothelial cells and leads to life-threatening complications such as left ventricular hypertrophy (LVH), renal failure, and stroke. Enzyme replacement therapy (ERT) results in Gb3 clearance; however, because of a short half-life in the body...
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