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Search Results - tejvir+khurana
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Site-blocking oligos (SBO) upregulate utrophin for treatment of Duchenne Muscular Dystrophy (DMD
Synthetic oligonucleotides blocking natural degradation of utrophin to treat muscular dystrophy. Problem: Duchenne Muscular Dystrophy (DMD) is a severe form of muscular dystrophy affecting males. A genetic mutation causes decreased production of dystrophin, a critical protein connecting cells to surrounding tissue. This leads to improper muscle...
Published: 6/20/2024
|
Inventor(s):
Tejvir Khurana
Keywords(s):
Genetic Disorders
,
Neurodegenerative Diseases
,
Orphan Disease
Category(s):
Technology Classifications > Therapeutics
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...
Published: 6/20/2024
|
Inventor(s):
Tejvir Khurana
,
Donna Huryn
Keywords(s):
Drug Target
,
Genetic Disorders
Category(s):
Technology Classifications > Therapeutics
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. Problem: Duchenne 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 which ultimately causes death...
Published: 6/20/2024
|
Inventor(s):
Tejvir Khurana
Keywords(s):
Gene Therapy
Category(s):
Technology Classifications > Therapeutics
Therapeutic approach for treatment of Duchenne Muscular Dystrophy
Technology Overview: 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...
Published: 2/12/2024
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Inventor(s):
Tejvir Khurana
Keywords(s):
Category(s):
Technology Classifications > Therapeutics