Local Dystrophin Restoration with Antisense Oligonucleotide PRO051

2007761 citationsJournal Article

Authors

Judith C. van Deutekom · Leiden University Medical Center

Anneke A. M. Janson · Leiden University Medical Center

Ieke B. Ginjaar · Leiden University Medical Center

Wendy S. Frankhuizen · Leiden University Medical Center

Annemieke Aartsma‐Rus · Leiden University Medical Center

Mattie Bremmer-Bout · Leiden University Medical Center

Johan T. den Dunnen

Abstract

Intramuscular injection of antisense oligonucleotide PRO051 induced dystrophin synthesis in four patients with Duchenne's muscular dystrophy who had suitable mutations, suggesting that further studies might be feasible.

Topics & Keywords

Muscle Physiology and Disorders RNA Interference and Gene Delivery Virus-based gene therapy research

Publication Details

Published in: New England Journal of Medicine

Volume 357, Issue 26, pp. 2677-2686

DOI: 10.1056/nejmoa073108

Field-Weighted Citation Impact: 30.52

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Local Dystrophin Restoration with Antisense Oligonucleotide PRO051

Authors

Abstract

Topics & Keywords

Publication Details