Scientists have managed to successfully transplant stem cells from patients with a rare form of muscular dystrophy into mice that suffered from the same form of dystrophy.
A new study published in Science Translational Medicine reveals that researchers have, for the first time, managed to turn fibroblast cells, i.e. common cells within connective tissue, from muscular dystrophy patients into stem cells and subsequently changed these cells into muscle precursor cells. After modifying the muscle precursor cells genetically, the researchers transplanted them into mice.
In future, this new technique could be used in order to treat patients with the rare condition of limb-girdle muscular dystrophy, which primarily affects the shoulders and hips, and maybe other types of muscular dystrophies. The method was initially developed in Milan at the San Raffaele Scientific Institute and was completed at UCL.
Muscular dystrophy is a genetic disorder, which typically affects skeletal muscles. The condition leads to severely impaired mobility and can, in severe cases result in respiratory and cardiac dysfunction. At present, there is no effective treatment for the condition. A number of new potential therapies, including cell therapy, are entering clinical trials.
The scientists of this study concentrated their research on genetically modifying mesoangioblasts, i.e. a self-renewing cell that originates from the dorsal aorta and differentiates into most mesodermal tissues, which demonstrated its potential for treating muscular dystrophy in earlier studies.
Given that the muscles of patients with muscular dystrophy are depleted of mesonangioblasts, the researchers were unable to obtain sufficient numbers of these cells from patients with limb-girdle muscular dystrophy, and therefore “reprogrammed” adult cells from these patients into stem cells, which enabled them to prompt them to differentiate into mesoangioblast-like cells. The team then genetically corrected these ‘progenitor’ cells by using a viral vector, and injected them into mice with muscular dystrophy so that the cells targeted damaged muscle fibers.
In a mice study, the same process demonstrated that dystrophic mice were able to run on a treadmill for longer a longer time than dystrophic mice that did not receive the cells.
Research leader, Dr Francesco Saverio Tedesco, from UCL Cell & Developmental Biology, who led the study, explained:
“This is a major proof of concept study. We have shown that we can bypass the limited amount of patients’ muscle stem cells using induced pluripotent stem cells and then produce unlimited numbers of genetically corrected progenitor cells.”
Professor Giulio Cossu, also an author at UCL, concluded:
“This technique may be useful in the future for treating limb-girdle muscular dystrophy and perhaps other forms of muscular dystrophy. This procedure is very promising, but it will need to be strenuously validated before it can be translated into a clinical setting, also considering that clinical safety for these “reprogrammed” stem cells has not yet been demonstrated for any disease.”
Written by Petra Rattue