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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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Identification of Small Molecule and Genetic Modulators of AON-Induced Dystrophin Exon Skipping by High-Throughput Screening
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Generation of human induced pluripotent stem cells from dermal fibroblasts
W. E. Lowry et al.
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Judith C. van Deutekom et al.
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Marcella Neri et al.
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Cellular senescence in human myoblasts is overcome by human telomerase reverse transcriptase and cyclin-dependent kinase 4: consequences in aging muscle and therapeutic strategies for muscular dystrophies
Chun-Hong Zhu et al.
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Chang-Hao Cui et al.
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J Alter et al.
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Calcium release from ryanodine receptors in the nucleoplasmic reticulum
P Marius et al.
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A Verrotti et al.
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S Kobayashi et al.
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Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing
P Sazani et al.
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Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse
QL Lu et al.
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Ryanodine receptor calcium release channels
M Fill et al.
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Recombinant micro-genes and dystrophin viral vectors
G Dickson et al.
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Dantrolene inhibition of ryanodine receptor Ca2+ release channels -: Molecular mechanism and isoform selectivity
FY Zhao et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2001)
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