— Technology Description
Researchers at Washington University in St. Louis have developed a 3D-printed device for the subcutaneous transplantation of stem cell-derived beta (SC-beta) cells for diabetes cell replacement therapy. Diabetes mellitus, a disease characterized by loss of blood glucose cont…
— Technology Description
Researchers at Washington University in St. Louis have developed an improved method to differentiate stem-cells into functionally mature, insulin-secreting beta cells (SC-beta cells). Pancreatic beta cells play an important role in controlling blood glucose levels as they se…
— Disease indication: metabolic disorders including insulin resistance and diabetic nephropathy
Drug format: chemically modified synthetic microRNA (miRNA)
Drug class: first-in-class for metabolic disease
Research stage and Preliminary data:
Target: miR-29 mimics can increase the gene expressio…
— Technology Description
Dr. Fumihiko Urano at Washington University in St. Louis (WUSTL) and colleagues have developed therapeutic strategies to treat Wolfram syndrome. Wolfram syndrome is a rare, life-threating, genetic disorder characterized by insulin-dependent diabetes mellitus, diabetes insipi…
— Lysosomal storage disorders result in severe, multi-system dysfunction and frequently, early death. Currently, a number of lysosomal storage disorders are treated with enzyme replacement therapy (ERT), which requires periodic infusions of the deficient enzyme. However, in a number of instances the…
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