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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-esclation, proof-of-concept study

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Academic year: 2020

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Figure 1: Deletions and predicted results of exon skipping in the patients who were studied(A) Pre-mRNA transcripts and dystrophin protein products from full length DMD, in patients with Duchenne muscular dystrophy, and predicted protein sequences after ex
Table 1: Baseline characteristics, exons targeted by PCR primers, and predicted amplicon sizes
Figure 2: Procedure for prescreening of patients before injection of AVI-4658. Patient 3 is shown as an example; similar results were obtained for all patients
Table 2: Safety studies and adverse reactions documented during the trial
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