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Stem cells for Duchenne?

Adult stem cells taken from humans suffering from Duchenne muscular dystrophy can be genetically modified and used to treat the disease in a mouse model, researchers linkurl:report;http://www.cellstemcell.com/ today in Cell Stem Cell. Duchenne muscular dystrophy is a progressive condition caused by a mutation on the X chromosome that leads to a lack of dystrophin protein in muscle. The mutation is usually caused by a deletion or mutation in the gene, leading to a shift in the reading frame of m

Alla Katsnelson
Adult stem cells taken from humans suffering from Duchenne muscular dystrophy can be genetically modified and used to treat the disease in a mouse model, researchers linkurl:report;http://www.cellstemcell.com/ today in Cell Stem Cell. Duchenne muscular dystrophy is a progressive condition caused by a mutation on the X chromosome that leads to a lack of dystrophin protein in muscle. The mutation is usually caused by a deletion or mutation in the gene, leading to a shift in the reading frame of mRNA translation. In past studies, injecting Duchenne mice with normal muscle cells has temporarily staved off disease symptoms, but that technique does not work reliably and can cause immune rejection. So researchers have linkurl:proposed;http://www.ncbi.nlm.nih.gov/sites/entrez?Db=PubMed&Cmd=ShowDetailView&TermToSearch=16691118&ordinalpos=3&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_RVDocSum delivering muscle progenitor cells instead. Yvan Torrente of the University of Milan, Italy, and colleagues took human blood- and muscle-derived stem cells from Duchenne patients. They used linkurl:antisense oligonucleotides;http://www.the-scientist.com/article/display/21438/ delivered by linkurl:lentiviral vectors;http://www.the-scientist.com/article/display/19298/ to mask the...

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