Optimizing gene therapy to help breathing in Duchenne muscular dystrophy

Who could benefit from this research

What this research studies

Duchenne muscular dystrophy damages muscles, including the diaphragm and respiratory muscles, leading to breathing problems. This project uses AAV-based gene therapy to deliver dystrophin-related material in mouse models that mimic human DMD, with a focus on the respiratory system. By studying how well breathing and the nerves controlling breathing respond in these models, the researchers aim to identify factors that could make future therapies safer and more effective for patients. The work will compare humanized mouse models to standard DMD models to better predict how a therapy might perform in people. If successful, the findings could guide the development of treatments that protect or restore respiratory function in DMD patients.

Why it matters

Where this research is happening

• Duke University
  Durham, United States Active

Researchers

• Principal investigator: Elmallah, Mai — Duke University
• Study coordinator: Elmallah, Mai

Related research

Other NIH-funded projects on the same topics.

• Improving Gene Therapy for Breathing Problems in Duchenne Muscular Dystrophy

  This research aims to make gene therapy better at helping children with Duchenne muscular dystrophy breathe more easily.

  NIH research Duke University · Durham, United States · NIH-11145834

• Improving gene therapy for breathing problems in Duchenne muscular dystrophy

  This study is looking at a new gene therapy to help improve breathing and muscle health for people with Duchenne muscular dystrophy (DMD) by using a special virus to deliver a helpful gene, and it's being tested in mouse models that mimic human conditions.

  NIH research Duke University · Durham, United States · NIH-10980431

• Advancing manufacturing and release testing for a Duchenne gene editing therapy

  A project to optimize how a dual-vector AAV gene editing treatment for Duchenne muscular dystrophy is made and tested, so it can move toward clinical use.

  NIH research Myogene Bio, LLC · San Diego, UNITED STATES · NIH-11526941

• Understanding why Duchenne Muscular Dystrophy worsens and how to stop it

  A research team at USC is studying Duchenne muscular dystrophy to find out why the disease continues to progress and how to interrupt harmful changes in muscle tissue.

  NIH research University of Southern California · Los Angeles, UNITED STATES · NIH-11502706

• Stopping steroid-related muscle and bone wasting in Duchenne muscular dystrophy

  Researchers are testing ways to protect muscles and bones in people with Duchenne muscular dystrophy who take long-term steroid medicines.

  NIH research Univ of Arkansas for Med Scis · Little Rock, United States · NIH-11458914

• MR clues to how nNOS affects energy use and blood flow in Becker and Duchenne muscular dystrophy

  This project looks at how advanced MRI tests can track muscle energy and blood flow changes in people with Becker and Duchenne muscular dystrophy as new gene therapies are explored.

  NIH research University of Florida · Gainesville, United States · NIH-11413866

About this research

1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
3. For full project details, budget, and progress reports, visit the official NIH RePORTER page.