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Local Dystrophin Restoration with Antisense Oligonucleotide PRO051
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Local Dystrophin Restoration with Antisense Oligonucleotide PRO051
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Journal Article
Local Dystrophin Restoration with Antisense Oligonucleotide PRO051
2007
Overview
This study explored the safety, tolerability, and dystrophin-restoring effect of a single, intramuscular dose of an antisense oligonucleotide, PRO051, to induce specific exon skipping during messenger RNA splicing and to restore dystrophin expression in patients with Duchenne's muscular dystrophy. Four patients, selected on the basis of mutational status, muscle condition, and a positive exon-skipping response to PRO051 in vitro, received the drug. Intramuscular injection of this compound induced local dystrophin synthesis.
This study explored the safety, tolerability, and dystrophin-restoring effect of an antisense oligonucleotide, PRO051, in patients with Duchenne's muscular dystrophy. Intramuscular injection of this compound induced local dystrophin synthesis.
Duchenne's muscular dystrophy is a severely debilitating childhood neuromuscular disease that affects 1 in 3500 newborn boys.
1
Progressive weakness of the skeletal muscles, cardiomyopathy, and respiratory failure are the most prominent features, but the brain can also be affected.
2
,
3
Virtually all patients are wheelchair-dependent by the age of 12 years, and most die in early adulthood. Improved ventilation techniques and glucocorticoid treatment have substantially improved fitness and muscle strength, prolonged mobility, and extended the expected lifespan from less than 20 years to 25 to 35 years.
4
–
6
However, there has been no treatment to prevent the eventual fatal outcome. . . .
Publisher
Massachusetts Medical Society
Subject
/ Child
/ Exons
/ Humans
/ Male
/ Muscular Dystrophy, Duchenne - drug therapy
/ Muscular Dystrophy, Duchenne - genetics
/ Muscular Dystrophy, Duchenne - metabolism
/ Mutation
/ Oligonucleotides - adverse effects
/ Oligonucleotides - therapeutic use
/ Oligonucleotides, Antisense - adverse effects
/ Oligonucleotides, Antisense - therapeutic use
/ Proteins
/ Rodents
