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Kyoto researchers show gene fix may work

OSAKA -- Kyoto University scientists have found that correcting a genetic defect that causes muscular dystrophy can alleviate the symptoms of the severest form of this debilitating disease.

     Akitsu Hotta and his colleagues used induced pluripotent stem (iPS) cells -- cells reprogrammed to an embryonic state -- to test their hypothesis. While much work remains to turn their discovery into a practical treatment, it does give cause for hope.

     Their findings appear Thursday in the online version of the journal Stem Cell Reports.

     Scientists have known that Duchenne muscular dystrophy, an incurable condition that leads to respiratory and cardiac failure, is the result of a genetic mutation that disrupts the production of a crucial protein.

     The Kyoto University team took iPS cells derived from patient tissue, removed the defective genetic sequence and replaced it with a normal one. When these cells turned into muscle cells, they produced the protein correctly. But iPS cells that did not undergo the genetic repair yielded muscle cells that soon degenerated.


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