Dogs with Duchenne Treated with Gene Therapy

Researchers restored muscle function in animals with muscular dystrophy.

By | July 25, 2017

PIXABAY, PAULICEK0

Like humans, some golden retrievers develop Duchenne muscular dystrophy (DMD), a hereditary muscle wasting condition that begins early in life. Using gene therapy, scientists were able to restore muscle function in dogs with the disease, according to a study published today (July 25) in Nature Communications.

Researchers injected microdystrophin, a shortened version of the dystrophin gene that individuals with DMD lack, into 12 dogs with the disease. The treatment led to improved muscle function in those animals for more than two years.

“This preclinical study demonstrates the safety and efficacy of microdystrophin, and makes it possible to consider developing a clinical trial in patients,” study coauthor Caroline Le Guiner of the Université de Nantes in France, says in a statement. “Indeed, this is the first time that it has been possible to treat the whole body of a large-sized animal with this protein.”

Scientists have also used CRISPR to correct the disease-causing mutations in mouse models of DMD and in the cells of a human patient with the condition.

See “CRISPR Corrects Duchenne-Causing Mutations

“This [study] is very encouraging, as current treatments for muscular dystrophy are merely palliative and patients are under constant medical care throughout their life,” John Counsell, a research associate at University College London who was not involved in the study, in a statement published by the Science Media Center. “Further preclinical trials will be required to show that this treatment can be effective in patients.”

Add a Comment

Avatar of: You

You

Processing...
Processing...

Sign In with your LabX Media Group Passport to leave a comment

Not a member? Register Now!

LabX Media Group Passport Logo

Popular Now

  1. 2017 Top 10 Innovations
    Features 2017 Top 10 Innovations

    From single-cell analysis to whole-genome sequencing, this year’s best new products shine on many levels.

  2. Thousands of Mutations Accumulate in the Human Brain Over a Lifetime
  3. Antiviral Immunotherapy Comes of Age
    News Analysis Antiviral Immunotherapy Comes of Age

    T-cell therapies are not just for cancer. Researchers are also advancing immunotherapy methods to protect bone marrow transplant patients from viral infections. 

  4. Search for Life on the Red Planet
FreeShip