That was enough for the muscle to carry some weight, and one injection
produced dystrophin for 3 months, the group reports in the 6 July online Nature Medicine.
Duchenne muscular dystrophy is caused by problems with the body's ability to
produce dystrophin, a long protein chain that binds the interior of a muscle fiber to its surrounding support structure.
Patients suffering from Duchenne muscular dystrophy are unable to
produce dystrophin.
Not exact matches
Once the UCLA researchers had
produced iPS cells that were free from Duchenne mutations, they differentiated the iPS cells into cardiac muscle and skeletal muscle cells and then transplanted the skeletal muscle cells into mice that had a genetic mutation in the
dystrophin gene.
Mice treated with antisense RNA (bottom)
produced more
dystrophin — colored red in this image — than did control mice (top).
In the current study, Dr. Rudnicki and his team discovered that muscle stem cells also express the
dystrophin protein, and without this protein, they
produce ten-fold fewer muscle precursor cells, which in - turn generate fewer functional muscle fibres.
Dr. Eric Olson (right) shows the
dystrophin protein (red)
produced in gene - edited heart muscle cells taken from a DMD patient's blood.
Children with DMD have a mutation that cripples the body's ability to
produce a protein called
dystrophin, which helps absorb the shock or energy that's created when a muscle contracts.
Once they were able to isolate skeletal muscle cells using the newly identified surface markers, the research team matured those cells in the lab to create
dystrophin -
producing muscle fibers.
Muscles from mice treated with modified human stem cells show human
dystrophin -
producing muscle fibers (yellow) integrated among mouse muscle fibers (red).