DMD Therapy Exondys 51 Gets a Hand from Immune Cells, Study Reports

DMD Therapy Exondys 51 Gets a Hand from Immune Cells, Study Reports: "The newly approved Duchenne muscular dystrophy therapy Exondys 51 (eteplirsen) stays in immune cells several days after leaving the blood, continuing to help regenerate muscle fibers, according to a study in mice."



The newly approved Duchenne muscular dystrophy therapy Exondys 51 (eteplirsen) stays in immune cells several days after leaving the blood, continuing to help regenerate muscle fibers, according to a study in mice.

CRISPR-Gold corrects gene mutations in mice with Duchenne muscular dystrophy

CRISPR-Gold corrects gene mutations in mice with Duchenne muscular dystrophy: "Scientists at the University of California, Berkeley, have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes Duchenne muscular dystrophy, a severe muscle-wasting disease. A new study shows that a single injection of CRISPR-Gold, as the new delivery system is called, into mice with Duchenne muscular dystrophy led to an 18-times-higher correction rate and a two-fold increase in a strength and agility test compared to control groups."



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