Open AccessLocal Dystrophin Restoration with Antisense Oligonucleotide PRO051
Author(s) -
Judith C. van Deutekom,
Anneke A. M. Janson,
Ieke B. Ginjaar,
Wendy S. Frankhuizen,
Annemieke AartsmaRus,
Mattie Bremmer-Bout,
Johan T. den Dunnen,
Klaas Koop,
Anneke J. van der Kooi,
Nathalie Goemans,
Sjef J. de Kimpe,
Peter F. Ekhart,
Edna H. Venneker,
Gerard Platenburg,
Jan J.G.M. Verschuuren,
GertJan B. van Ommen
Publication year - 2007
Publication title -
new england journal of medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 19.889
H-Index - 1030
eISSN - 1533-4406
pISSN - 0028-4793
DOI - 10.1056/nejmoa073108
Subject(s) - dystrophin , duchenne muscular dystrophy , exon skipping , exon , medicine , muscular dystrophy , utrophin , microbiology and biotechnology , mdx mouse , messenger rna , alternative splicing , biology , genetics , gene
Duchenne's muscular dystrophy is associated with severe, progressive muscle weakness and typically leads to death between the ages of 20 and 35 years. By inducing specific exon skipping during messenger RNA (mRNA) splicing, antisense compounds were recently shown to correct the open reading frame of the DMD gene and thus to restore dystrophin expression in vitro and in animal models in vivo. We explored the safety, adverse-event profile, and local dystrophin-restoring effect of a single, intramuscular dose of an antisense oligonucleotide, PRO051, in patients with this disease.
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