Apr. 24, 2020 — Children born with Duchenne muscular dystrophy have a mutation in the X-chromosome gene that would normally code for dystrophin, a protein that provides structural integrity to skeletal muscles. The ...
Feb. 24, 2020 — Testing of small molecules in mouse models for Duchenne muscular dystrophy shows promise for restoration of muscle structure and ...
Dec. 9, 2019 — Repairing faulty genes to prevent and cure disease is something researchers have been working towards for many years. While Class 2 CRISPR systems show great promise as gene editing tools in human ...
Sep. 25, 2019 — A new therapeutic being tested is showing early promise as a more effective treatment that could help nearly half of patients with Duchenne muscular dystrophy (DMD). The treatment -- a cocktail of ...
Sep. 24, 2019 — A new clinical trial found a cost-effective generic drug works just as well as a more expensive drug in preserving heart function in boys with Duchenne muscular ...
Sep. 17, 2019 — Duchenne muscular dystrophy (DMD) is a rare but devastating genetic disorder that causes muscle loss and physical impairments. Researchers have shown in a mouse study that the powerful gene editing ...
July 18, 2019 — A team has identified a gene that could make immunotherapy treatments, specifically checkpoint inhibitors, work for a wider variety of cancer patients. The study found that when the DUX4 gene is ...
July 9, 2019 — Duchenne muscular dystrophy occurs in boys and is characterized by progressive muscle degeneration and weakness leading to a decline in respiratory function. Strategies to arrest this severe ...
June 14, 2019 — Researchers found that the protein sarcospan can play a major role in combating heart failure in patients with Duchenne muscular ...
June 7, 2019 — Muscle decline caused by ageing and certain diseases could be dramatically slowed by stopping a chain reaction that damages cells, new research ...
June 3, 2019 — Researchers have found that lithium improves muscle size and strength in mice with a rare form of muscular dystrophy that causes weakness in the shoulders and hips. The findings could lead to a drug ...
Apr. 17, 2019 — Scientists have uncovered a molecular signaling pathway involving Stat3 and Fam3a proteins that regulates how muscle stem cells decide whether to self-renew or differentiate -- an insight that could ...
Apr. 10, 2019 — Scientists have developed a strategy for editing and repairing a particular type of genetic mutation associated with microduplications using CRISPR/Cas9 and a seldom-used DNA repair pathway. This ...
Apr. 1, 2019 — The compound called Cugamycin works by recognizing toxic RNA repeats and destroying the garbled gene ...
Mar. 12, 2019 — A research team how a type of protein that is embedded in the inner nuclear membrane clears out of the system once it has served its ...
Mar. 6, 2019 — Scientists have developed a method to boost the efficiency of CRISPR gene editing in Duchenne muscular dystrophy (DMD), according to a study that could have implications for optimizing gene therapies ...
Feb. 27, 2019 — By sequencing the entire genomes of tumor cells from six people with a rare cancer of the nose and sinus cavity, researchers report they unexpectedly found the same genetic change -- one in a gene ...
Feb. 21, 2019 — Patients with Duchenne muscular dystrophy (DMD) have few treatment options. Medications currently available or in development either target only a subset of DMD patients with a particular genetic ...
Feb. 13, 2019 — The University of Minnesota Medical School continues its legacy of advancing cell replacement therapies with a scientific breakthrough that highlights the promise of cell therapies for muscular ...
Feb. 11, 2019 — Prednisone, the current standard of care used to treat kids with Duchenne muscular dystrophy (DMD), reduces chronic inflammation but has harsh side effects. Eplerenone, a heart failure drug, is used ...
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