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Fighting sickle cell disease by looking back to babyhood
Fast-track research focused on developing a new gene-modifying sickle cell disease treatment at the Medical University of South Carolina could lead to human clinical trials in as few as three years.
Patrick Woster, the SmartState endowed chair in medicinal chemistry at MUSC, said the project takes aim at the enzyme lysine-specific demethylase 1, or LSD 1. That enzyme is part of the biological process that makes mutated hemoglobin, leading to sickle-shaped blood cells. They stick together, causing extremely painful and dangerous blood flow problems. Inhibiting LSD 1, making it less effective, could turn an important gene back on again and keep sickle-shaped cells from sticking together.
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.