Why the study?
Does transfection with antisense oligoribonucleotides induce dystrophin expression and exon skipping in cultured mdx muscle cells?
Population
Primary mdx myoblasts in culture (mdx mouse model of Duchenne muscular dystrophy)
Design
Preclinical
Authors
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Provides proof-of-concept for splice modulation in mdx cells; hypothesis-generating for Duchenne muscular dystrophy and should not yet change practice.
Does transfection with antisense oligoribonucleotides induce dystrophin expression and exon skipping in cultured mdx muscle cells?
Antisense oligoribonucleotides can induce exon skipping and restore dystrophin expression in mdx muscle cells, offering a potential therapeutic approach for Duchenne muscular dystrophy.
Dunckley et al. (1998) studied this question.
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