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Stem Cell Therapy May Cure Cystinosis

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Aired 9/18/09

San Diego researchers say they have used stem cells to cure a rare but deadly disease in lab animals.

— San Diego researchers say they have used stem cells to cure a rare but deadly disease in lab animals.

Scientists at the Scripps Research Institute were targeting a disease called cystinosis. The disease occurs in children when a cell byproduct, cystine, cannot be discarded by cells due to a genetic disorder. This results in a host of diseases that include diabetes and kidney failure.

Scripps researchers took stem cells taken from bone marrow and put them into lab mice. They found the stem cells either replaced or fused with the defective cells in every body tissue, and that cured the mice. Stephanie Cherqui, the principal investigator of the research, says stem cells normally target one organ, and experts didn't expect success with a multi-systemic disease like cystinosis.

"Fortunately I did this experiment anyway," she said. "And even me, I was very surprised."

Cherqui says the next step would be human clinical trials.

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