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 ...

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 ...

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 ...

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 ...

New hope for Duchenne muscular dystrophy (DMD) patients. A mouse genetic study in PLoS Medicine reports that targeting the P2RX7 gene, a purinoreceptor, may halt the progression of DMD. Duchenne ...

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 ...

Boston, Massachusetts--(Newsfile Corp. - April 29, 2025) - Tevard Biosciences, Inc., a privately held biotechnology company pioneering tRNA-based therapies to cure a broad range of genetic diseases, ...

Researchers from Kyoto University demonstrate how a dual CRISPR RNA method restored dystrophin protein function in induced pluripotent stem cells derived from Duchenne muscular dystrophy (DMD) ...

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 ...