Source: ISNA, Tehran
Iranian researcher and lecturer Radbod Darabi jointly with his collogues from the University of Minnesota's Lillehei Heart Institute have effectively treated muscular dystrophy in mice using human stem cells derived from a new process which for the first time makes the production of human muscle cells from stem cells efficient and effective.
Radbod Darabi, MD, PhD with Rita Perlingeiro, PhD.
(Credit: Image courtesy of University of Minnesota Academic Health Center)
The research outlines the strategy for the development of a rapidly dividing population of muscle-forming cells derived from induced pluripotent (iPS) cells.
IPS cells have all of the potential of embryonic stem (ES) cells, but are derived by reprogramming skin cells. They can be patient-specific, which renders them unlikely to be rejected, and do not involve the destruction of embryos.
This is the first time that human stem cells have been shown to be effective in the treatment of muscular dystrophy.
According to the researchers, there has been a significant lag in translating studies using mouse stem cells into therapeutically relevant studies involving human stem cells.
This lag has dramatically limited the development of cell therapies or clinical trials for human patients.
The latest research from the University of Minnesota provides the proof-of-principle for treating muscular dystrophy with human iPS cells, setting the stage for future human clinical trials.
As the researchers noted one of the biggest barriers to the development of cell-based therapies for neuromuscular disorders like muscular dystrophy has been obtaining sufficient muscle progenitor cells to produce a therapeutically effective response.
They stressed that up until now, deriving engraftable skeletal muscle stem cells from human pluripotent stem cells hasn't been possible. The results demonstrate that it is indeed possible and sets the stage for the development of a clinically meaningful treatment approach.
Related Article: Human ES- and iPS-Derived Myogenic Progenitors Restore DYSTROPHIN and Improve Contractility upon Transplantation in Dystrophic Mice - Cell Stem Cells
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