eLife: Significant findings have been made in the study of fatal muscular dystrophy diseases

Doo's muscular dystrophy (DMD) is caused by a genetic mutation that affects one in every 5,000 boys born
.

Children with this condition require a wheelchair as a teenager, and most will die
in their 30s or 30s.

Previously, it was widely believed that DMD began with muscle fibers – cells involved in contraction that make up the body
of all muscles.

New research suggests that the disease begins earlier
in cells destined to become muscle fibers, known as myoblasts.

The study, published in eLife, is part of an ongoing collaboration between scientists from the University of Portsmouth, the French National Centre for Scientific Research, I-STEM, AFM France and the main pharmacological institutes of the Polish Academy of Sciences

Professor Darek Gorecki, senior author from the University of Portsmouth's School of Pharmacy and Biological Sciences, said: "These findings are significant because they change the way
we understand this disease.

"Because these myogenic cells are dysfunctional, damaged muscles cannot be repaired
effectively.

Last year, the team released the results of DMD modeling, looking at its development in terms of
its initial triggers and initial performance.

Professor Gorecki added: "At the moment, we are targeting the late stages of the disease, treating teenage patients at a time when muscle degeneration has already caused them harm
.

"Conversely, if we try to correct cells at the beginning of the pathological process, we may be able to delay muscle degeneration and extend the patient's lifespan
.

The paper says the new technology could be the key
to producing effective therapies for this devastating disease.

essay

Loss of full-length dystrophin expression results in major cell-autonomous 2 abnormalities in proliferating myoblasts