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BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2009)
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BLOOD (2009)
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HUMAN MUTATION (2009)
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. (vol 8, pg 918, 2009)
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HUMAN MOLECULAR GENETICS (2008)
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JOURNAL OF BIOLOGICAL CHEMISTRY (2008)
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MOLECULAR THERAPY (2008)
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PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)
Local dystrophin restoration with antisense oligonucleotide PRO051
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NEW ENGLAND JOURNAL OF MEDICINE (2007)
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BLOOD (2007)
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GENE THERAPY (2007)
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PTC124 targets genetic disorders caused by nonsense mutations
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NATURE MEDICINE (2003)
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HUMAN MOLECULAR GENETICS (2003)
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