Abstract: Molecules are provided for inducing or facilitating exon skipping in forming spliced mRNA products from pre-mRNA molecules in cells. The molecules may be provided directly as oligonucleotides or expression products of vectors that are administered to a subject. High rates of skipping can be achieved. High rates of skipping reduce the severity of a disease like Duchene Muscular Dystrophy so that the disease is more like Becker Muscular Dystrophy. This is a severe reduction in symptom severity and mortality.
Type:
Grant
Filed:
September 10, 2009
Date of Patent:
December 27, 2011
Assignee:
Royal Holloway and Bedford New College Royal Holloway, University of London
Inventors:
Linda Popplewell, Ian Graham, John George Dickson