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Gene therapy for treatment of Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy (DMD) therapy deletes let-7c miRNA binding sites using CRISPR-Cas9 based genome editing and AAV based delivery.Inventor Tejvir Khurana, MD, PhDProblemDuchenne Muscular Dystrophy (DMD) is a fatal disease characterized by progressive muscle degeneration and weakness that affects 1 in 3,500 live-born males worldwide, and wh...
Published: 8/2/2018   |   Inventor(s): Tejvir Khurana
Keywords(s):  
Category(s): Therapeutics & Vaccines
Therapeutic Approach for Treatment of Duchenne Muscular Dystrophy
Technology: Duchenne Muscular Dystrophy (DMD) is the most common X-linked fatal neuromuscular disorder, affecting approximately 1 in every 3500 male births. It is characterized by progressive degeneration of skeletal and cardiac muscle due to a defective form of the gene dystrophin. There is currently no cure for DMD and the available treatment op...
Published: 7/17/2018   |   Inventor(s): Tejvir Khurana
Keywords(s):  
Category(s): Therapeutics & Vaccines
SMALL MOLECULE UTROPHIN UP-REGULATORS FOR TREATMENT OF MUSCULAR DYSTROPHY
Duchenne muscular dystrophy (DMD) is a recessive X-linked form of muscular dystrophy affecting 1 in 3,600 boys and is caused by mutations in the dystrophin gene. DMD leads to progressive muscle degeneration and eventually death. Many strategies have been proposed and are being studied for treatment of DMD and other more rare dystrophinopathies; h...
Published: 1/31/2018   |   Inventor(s): Tejvir Khurana, Catherine Moorwood
Keywords(s):  
Category(s): Therapeutics & Vaccines