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                           JeffNEWS, July 1, 1995
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Jefferson Researchers Find New Genetic Treatment
for Bone and Cartilage Disease 
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Researchers at Jefferson Medical College hope they have found a simple way 
around one barrier to therapy of genetic diseases of bone and cartilage.

A new procedure developed by the Jefferson researchers could pave the way 
for gene therapy for brittle bone disease and dwarfism. In the future, it
may well pave the way for therapy of osteoporosis and osteoarthritis. The 
procedure was described in the May 22 issue of Proceedings of the National 
Academy of Sciences.

The research, currently in the laboratory stage, shows promise that taking 
bone marrow cells which are "precursors" to bone and cartilage cells from a 
healthy person and transplanting them, using a procedure that is more 
sophisticated but similar to a blood transfusion, through the bloodstream to 
a patient with severe bone or cartilage disease caused by a genetic defect 
may be effective therapy. The study indicates that transplanting the 
"precursor" cells will overcome such patients' inability to manufacture their 
own healthy bone and cartilage within one to five months.

The research team headed by Darwin J. Prockop, MD, PhD, director of the 
Jefferson Institute of Molecular Medicine, includes Michael D. O'Hara, PhD, 
assistant professor of radiation oncology and nuclear medicine; Dennis B. 
Leeper, PhD, professor of radiation oncology and nuclear medicine; Mr. 
Kenneth Halford and Ruth F. Pereira, PhD, both of the Jefferson Institute of 
Molecular Medicine; and Marea D. Pollard, laboratory assistant.

For many years, scientists have been working to find ways to administer a 
normal gene to a patient so that the normal gene will either replace or 
effectively counteract the defective gene in all the appropriate cells of the 
body, thus curing the patient of the disease. 

Until now, using bone and cartilage precursor cells to treat generalized 
diseases of bone and cartilage seemed impractical, since these cells are 
widely dispersed and embedded in hard extracellular fibers and mineral, 
making the cells extremely difficult to isolate. In this new procedure 
suggested by the Jefferson research, bone and cartilage precursor cells are 
readily obtained from a donor with a needle and syringe. In a few days, they 
begin to grow very rapidly in vitro, making them ideal candidates for 
insertion into diseased patients. This is a major reason why the Jefferson 
research is so significant and promising for the future.


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