Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored dystrophin protein ...
Which do you think would win in a fight between gene editing and gene therapy? Well, we might find out sooner than expected, because Vertex Pharmaceuticals (NASDAQ: VRTX) has doubled down on a ...
The 3′-untranslated region (3′UTR) of some vertebrate dystrophin genes shows an extraordinary degree and extent of conservation (better than that of many coding regions), a phenomenon that remains ...
Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window BOSTON -- A ...
People with Duchenne muscular dystrophy carry a mutation in the DMD gene coding for dystrophin, a protein crucial for proper muscle function. Scientists are working to treat the disease at its genetic ...
Dyne’s exon-skipping therapy zeleciment rostudirsen resulted in an approximately sevenfold increase in dystrophin levels at six months and elicited functional improvements that are the “best ever” for ...
Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new ...
Researchers have found that if you have low levels of the DOT1L enzyme, you could be at risk for some types of heart disease. Everyone knows chocolate is critical to a happy Valentine's Day. Now ...
Vertex Pharmaceuticals recently doubled down on its gene editing collaboration with CRISPR Therapeutics, but it's miles behind an arguably simpler solution. DMD is a rare inherited muscle-wasting ...
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