A researcher has discovered muscle cells that can be transplanted into mice without triggering an immune reaction, raising the hope that simple cell transplants could one day treat lethal diseases in humans such as Duchenne dystrophy.
Johnny Huard of the University of Pittsburgh presented the results of a series of mouse experiments this week at the annual meeting of the American Society for Cell Biology in Washington, D.C.
Duchenne dystrophy is a genetic disease of boys in which the lack of the protein dystrophin leads to progressive, widespread muscle damage. Patients usually are in wheelchairs by age 12 and die in their 20s because their heart and respiratory muscles fail.
Other researchers have studied donor muscle cell transplantation as a treatment for Duchenne disease, but the strategy didn't work, partly because the recipient's immune system rejected the foreign cells.
Huard's team extracted stem cells from healthy mouse muscles and grew them in the lab. Stem cells are able to produce new cells in the body.
When the stem cells were injected into the muscles of Duchenne mice, they made muscle cells that contained dystrophin. The stem cells produced better results than transplanting more mature muscle cells.
In part that's because Huard's stem cells do not have the surface proteins that normally wave a red flag in front of the recipient's immune system and incite it to attack. Another reason is that the stem cells are able to renew themselves and, as Huard put it, make "baby stem cells" in the diseased muscle.
"We want to use normal cells that we don't have to genetically engineer," Huard said. "If it all turns out to be true in humans, maybe you can have a single donor and you can inject 50 different recipients with the same cells. That's going to be amazing."
The findings are being considered for publication next year in the Journal of Cell Biology.