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The gene that causes Duchenne muscular dystrophy can be repaired

15 April 2010 Email This Post Email This Post Print This Post Print This Post

Muscle System BackThe gene that causes Duchenne muscular dystrophy 1), a hereditary disease affecting one in 3500 males, can be repaired according to researchers from Universite Laval’s Faculty of Medicine and the CHUQ Research Center.

Researchers performed in vitro tests by inserting into human muscle cells a variety of meganucleases, enzymes with the ability to correct the dystrophin gene, and also in vivo with mice carrying the mutation that causes the illness. Both series of testing showed that the meganucleases can lead to a restoration of the normal nucleotide sequences of the dystrophin gene and its expression in muscle cells.

A number of hurdles must be overcome before this approach can be tested in humans, cautions Dr. Jacques P. Tremblay who led the team of researchers. “It must first be proven in laboratory animals that it is possible to insert a meganuclease targeting the dystrophin gene directly into muscle cells, and that this will induce the synthesis of dystrophin able to attach to the muscle fiber membrane,” explains the researcher. “We’re still two to three years away from this stage,” he estimates. “Subsequent stages, including human trials, could take even longer,” adds Dr. Tremblay.

1 Duchenne muscular dystrophy is characterized by a rapid progression of muscle degeneration that begins in early childhood leading to loss of ambulation and even death.

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