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Stem cells effective against rare bone disorder in trial with mice

People with osteogensis imperfecta, a genetic disorder, may benefit from bone marrow transplant, study finds

By Brian P. Dunleavy
A bone marrow transplant may help those with the rare bone disorder osteogensis imperfecta. Photo by Dr. Manuel González Reyes/Pixabay
A bone marrow transplant may help those with the rare bone disorder osteogensis imperfecta. Photo by Dr. Manuel González Reyes/Pixabay

Dec. 24 (UPI) -- Those with weak and brittle bones caused by a rare genetic disorder have reason for new hope with the discovery of a cell transplant treatment that may finally resolve the underlying cause of their disease.

In an article published Tuesday in the journal STEM CELLS, researchers show how they transplanted healthy donor bone marrow cells directly into the femur of mice with osteogensis imperfecta, or OI, a rare disease that affects bone strength.

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One month following transplantation, the researchers found 18 percent of the surface that was injected with the donor cells expressed osteoblasts -- cells that help form new bone tissue -- an indication of engraftment, or growth.

In addition, long-term engraftment was then observed at three and six months post-transplantation.

"This is a basic research study with potential for future translation into practice," study co-author Ivo Kalajzic, a researcher at the Center for Regenerative Medicine and Skeletal Development at the University of Connecticut, said in a statement.

Kalajzic and his colleagues also found that healthy donor cells replacing mutant collagen have the ability to improve bone strength and structure in transplant recipients.

OI are genetic disorders that mainly affect the bone. Patients with OI have bones that break easily, sometimes with no obvious cause.

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In recent years, research has shown that the disorders are caused by genetic mutations associated with type 1 collagen molecules. These molecules help the body process collagen, and damage to them results in defective bone structure.

Currently, treatments for OI attempt to correct the defective bone structure, but don't focus on the underlying collagen defect.

The new findings demonstrate, however, that healthy donor stem cells that produce normal collagen in OI patients have the potential to increase bone mass and correct the collagen defect causing the condition.

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