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Up-regulators of utrophin expression for the treatment of muscular dystrophy
Small molecules targeting utrophin mRNA untranslated regions overcome post-transcriptional repression Problem: Duchenne Muscular Dystrophy (DMD) is an X-linked neuromuscular disorder that affects approximately 1 in 3500 males worldwide. DMD is caused by mutations in the DMD gene that lead to severe reduction or loss of dystrophin protein. Without appropriate...
Published: 4/28/2020   |   Inventor(s): Tejvir Khurana, Donna Huryn
Keywords(s): Drug Target, Genetic Disorders, Musculoskeletal
Category(s): Therapeutics & Vaccines