Gene Therapy
Investigators at University of Western Australia target gene therapy
Published in Gene Therapy Weekly, February 1st, 2007
New research, "Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD," is the subject of a report. "Manipulation of pre-mRNA splicing by antisense oligonucleotides (AOs) offers considerable potential for a number of genetic disorders. One of these is Duchenne muscular dystrophy (DMD), where mutations in the dystrophin gene typically result in premature termination of translation that causes a loss of functional protein," scientists writing in the journal Gene Therapy report.
"AOs can induce exon skipping such that the mutation is by-passed and the reading frame restored, producing an internally deleted...
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Source: Gene Therapy Weekly (2007-02-01)
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