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BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2007)
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NATURE REVIEWS MOLECULAR CELL BIOLOGY (2006)
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NATURE BIOTECHNOLOGY (2006)
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JOURNAL OF MOLECULAR BIOLOGY (2006)
Trehalose reduces aggregate formation and delays pathology in a transgenic mouse model of oculopharyngeal muscular dystrophy
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MOLECULAR THERAPY (2006)
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NATURE REVIEWS GENETICS (2005)
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Z Wang et al.
NATURE BIOTECHNOLOGY (2005)
Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury
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MOLECULAR THERAPY (2005)
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
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SCIENCE (2004)
Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1
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BLOOD (2004)
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NUCLEIC ACIDS RESEARCH (2003)
Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
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MOLECULAR GENETICS AND METABOLISM (2003)
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