muscular dystrophy stem cell research, Early studies in the US successfully transplanted
stem cells from healthy newborn mice into muscles of mice bred to
simulate muscular dystrophy. These cells provide the missing protein and
are fully incorporated into muscle tissue.
This basic research contributed in part to the
establishment in 2004 in Canada of the first clinical trial for the
treatment of Duchenne muscular dystrophy. The results of this trial
demonstrated that when muscle precursor cells (called "myoblasts") from a
healthy donor are transplanted into the affected muscle of a patient
with Duchenne muscular dystrophy they fuse with the host myofibers and
donate their healthy genome, allowing the correct form of the gene to be
expressed. It is now known that a small fraction of healthy nuclei can
provide enough dystrophin to completely cure a myofiber and ensure its
survival.
The success of this cell transplantation protocol
depends on suppressing the immune system with a new drug called
Tacrolimus, on the injection of a high number of cells and on a short
distance between the injection sites. In other words, the effects are
localized to the site of cell injection. The limitation of this
technique is that cells have to be injected into each muscle in turn,
whereas muscular dystrophy affects the entire body.
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