DALLAS – April 30, 2021 – UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients. The approach, described online today in the journal Science Advances, could lead to a treatment for DMD and inform the treatment of other inherited diseases.
DALLAS – Dec. 21, 2020 – A UT Southwestern research team has catalogued gene activity in the skeletal muscle of mice, comparing healthy animals to those carrying a genetic mutation that causes Duchene muscular dystrophy (DMD) in humans. The findings, published online recently in PNAS, could lead to new treatments for this devastating degenerative disease and insights into factors that affect muscle development.
DALLAS – Nov. 4, 2020 – A mutation in the gene that causes cystic fibrosis may accelerate heart function decline in those with Duchenne muscular dystrophy (DMD), a new study by UT Southwestern researchers suggests. The findings, published online recently in the Journal of the American Heart Association, could help doctors develop new strategies to preserve heart function in this population, potentially extending patients’ lives.
Scientists at Sanford Burnham Prebys, Fondazione Santa Lucia IRCCS, and Università Cattolica del Sacro Cuore in Rome have shown that pharmacological (drug) correction of the content of extracellular vesicles released within dystrophic muscles can restore their ability to regenerate muscle and prevent muscle scarring. The study, published in EMBO Reports, reveals a promising new therapeutic approach for Duchenne muscular dystrophy (DMD), an incurable muscle-wasting condition.
A Rutgers-led team may have found the key to preventing Duchenne muscular dystrophy (DMD)-related heart disease, the leading cause of death in patients living with the disease
A new clinical trial conducted at The Ohio State University Wexner Medical Center found a cost-effective generic medication works just as well as a more expensive drug in preserving cardiovascular function in boys with Duchenne muscular dystrophy (DMD).