Muscular dystrophy, which affects approximately 250,000 people in the U.S., occurs when damaged muscle tissue is replaced with fibrous, fatty or bony tissue and loses function. For years, scientists have searched for a way to successfully treat the most common form of the disease, Duchenne Muscular Dystrophy (DMD), which primarily affects boys. Now, a team of University of Missouri researchers have successfully treated dogs with DMD and say that human clinical trials are being planned in the next few years.
“This is the most common muscle disease in boys, and there is currently no effective therapy,” saidDongsheng Duan, the study leader and the Margaret Proctor Mulligan Professor in Medical Research at the MU School of Medicine. “This discovery took our research team more than 10 years, but we believe we are on the cusp of having a treatment for the disease.”
Patients with Duchenne muscular dystrophy have a gene mutation that disrupts the production of a protein known as “dystrophin.” Absence of dystrophin starts a chain reaction that eventually leads to muscle cell degeneration and death. Affected boys lose their ability to walk and breathe as they get older. This places significant limitations on individuals afflicted with the disease. Dystrophin also is one of the largest genes in the human body.
“Due to its size, it is impossible to deliver the entire gene with a gene therapy vector, which is the vehicle that carries the therapeutic gene to the correct site in the body,” Duan said. “Through previous research, we were able to develop a miniature version of this gene called a microgene. This minimized dystrophin protected all muscles in the body of diseased mice.”
However, it took the team more than 10 years to develop a strategy that can safely send the micro-dystrophin to every muscle in a dog that is afflicted by the disease. The dog has a body size similar to that of an affected boy. Success in the dog will set the foundation for human tests.
In this latest study, the MU team demonstrated for the first time that a common virus can deliver the microgene to all muscles in the body of a diseased dog. The dogs were injected with the virus when they were two to three months old and just starting to show signs of DMD. The dogs are now six to seven months old and continue to develop normally.
“The virus we are using is one of the most common viruses; it is also a virus that produces no symptoms in the human body, making this a safe way to spread the dystrophin gene throughout the body,” Duan said. “These dogs develop DMD naturally in a similar manner as humans. It’s important to treat DMD early before the disease does a lot of damage as this therapy has the greatest impact at the early stages in life.”
The Latest on: Muscular Dystrophy
via Google News
The Latest on: Muscular Dystrophy
- Duchenne UK and Parent Project Muscular Dystrophy Award $350,000 to Address Immunological Challenges of Gene Therapy in Duchenne Muscular Dystrophyon February 18, 2021 at 7:00 am
Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), ...
- Duchenne muscular dystrophyon February 18, 2021 at 12:59 am
Duchenne muscular dystrophy is an X-linked progressive, muscle-wasting disease that manifests in childhood as difficulties with movement. This Primer by Aartsma-Rus and colleagues discusses the ...
- Muscular Dystrophy Association to Roll Out the 39th Annual MDA Shamrocks Campaign with Retailers Nationwideon February 16, 2021 at 9:31 pm
The Muscular Dystrophy Association (MDA) today launched the nation’s largest St. Patrick’s Day fundraising program, the 39th annual MDA Shamrocks campaign. Thousands of retailers nationwide are ...
- SC teacher with muscular dystrophy says returning to classroom without vaccine could be deadlyon February 15, 2021 at 9:57 pm
Lopez said she requested to teach virtually, but because this was denied she’s now on unpaid leave through the Family Medical Leave Act.
- Global Duchenne Muscular Dystrophy Therapeutics Market 2020 Segmentation Analysis, Key Players, Industry Share and Forecast by 2025on February 9, 2021 at 5:46 pm
MarketsandResearch.biz is aimed at offering market research study on Global Duchenne Muscular Dystrophy Therapeutics Market 2020 by Company, Type and Application, Forecast to 2025, provides an ...
- Parent Project Muscular Dystrophy Receives $100,000 Award from Duchenne Research Fund to Address Neurobehavioral Issues in Duchenneon February 9, 2021 at 7:00 am
Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), today announced sponsorship from the Duchenne ...
- Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophyon February 9, 2021 at 2:45 am
Duchenne muscular dystrophy (DMD) is an X-linked progressive muscle-wasting disorder that is caused by a lack of functional dystrophin, a cytoplasmic protein necessary for the structural integrity of ...
- United States Duchenne Muscular Dystrophy Market and Competitive Landscape Report 2021 - ResearchAndMarkets.comon February 3, 2021 at 9:16 am
The “US Duchenne Muscular Dystrophy Market and Competitive Landscape - 2021” report has been added to ResearchAndMarkets.com’s offering. US Duchenne Muscular Dystrophy Market and Competitive Landscape ...
- Muscular Dystrophy Association Announces Nyheim Hines as 2021 National Spokespersonon February 3, 2021 at 4:14 am
NEW YORK, Jan. 27, 2021 /PRNewswire/ -- The Muscular Dystrophy Association (MDA) announced Nyheim Hines as its 2021 National Spokesperson to raise awareness and funds for the not-for-profit ...
- Muscular Dystrophy Symptomson February 1, 2021 at 4:00 pm
The muscle weakness that occurs in all different types of muscular dystrophy shares certain characteristics. First of all, it is most often symmetrical, i.e. the weakness on both sides of the body ...
via Bing News