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Treatment May Correct Gene Defect in Muscular Dystrophy

Injection of antisense oligonucleotide induces dystrophin production

WEDNESDAY, Dec. 26 (HealthDay News) -- An antisense oligonucleotide can correct the genetic defect in patients with Duchenne's muscular dystrophy and induce dystrophin production, according to a report in the Dec. 27 issue of the New England Journal of Medicine.

Judith C. van Deutekom, Ph.D., from Leiden University Medical Center in the Netherlands, and colleagues injected an antisense oligonucleotide, PRO051, to induce exon skipping in the DMD gene in four patients with Duchenne's muscular dystrophy. Patients were chosen based on mutational status.

After 28 days, the researchers found that all patients showed skipping of exon 51 and sarcolemmal dystrophin in 64 percent to 97 percent of muscle fibers. Dystrophin comprised 3 percent to 12 percent of the total protein compared to that present in control muscle. There were no adverse events associated with treatment.

"Intramuscular injection of antisense oligonucleotide PRO051 induced dystrophin synthesis in four patients with Duchenne's muscular dystrophy who had suitable mutations, suggesting that further studies might be feasible," van Deutekom and colleagues conclude.

The study was partially sponsored by Prosensa B.V.

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