Article
Improved antisense oligonucleotide induced exon skipping in the <i>mdx</i> mouse model of muscular dystrophy
13 Aug 2002
Abstract excerpt
BACKGROUND: Duchenne muscular dystrophy (DMD) is a fatal genetic disorder caused by dystrophin gene mutations that preclude synthesis of a functional protein. One potential treatment of the disorder has utilised antisense oligoribonucleotides (AOs) to induce removal of disease-associated exons during pre-mRNA processing. Induced in-frame mRNA transcripts encode a shorter but functional dystrophin. We have...
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