Local dystrophin restoration with antisense oligonucleotide PRO051.
By J. V. van Deutekom, A. Janson, I. Ginjaar, W. Frankhuizen, A. Aartsma-Rus, M. Bremmer-Bout, J. D. den Dunnen, K. Koop, Anneke J van der Kooi, N. Goemans and 6 more. New England Journal of Medicine, 2007
813 citations recorded by the source corpus.
Subfields: Cellular and Molecular Neuroscience, Molecular Biology
View Paper: https://www.semanticscholar.org/paper/2bc50a9d83f99e9784c39479dfc56280a096d2a8
How researchers see this paper
The researchers whose trusted networks rank it highest, strongest advocate first. Every researcher gets a different order.
In Molecular Biology
Citations in the corpus
- RNA therapeutics: Beyond RNA interference and antisense oligonucleotides (2012, methodology): “The latter two SSO chemistries have been used in clinical trials that tested splicing modulation as a treatment for DM”
- Making muscle: skeletal myogenesis in vivo and in vitro (2017, background): “…models can now be generated for a large spectrum of muscle pathologies (Avior et al., 2016;Merkle and Eggan, 2013; Rüegg and Glass, 2011), facilitating the development of in vitro therapeutic approaches (Kazuki et al., 2010; Rohwedel et al., 2001; Tran et al., 2013; van Deutekom et al., 2007).”
- Cell-penetrating peptides: design, synthesis, and applications. (2014, background): “skipping, splice-switching oligonucleotides efficiently restored dystrophin production.(183) The most common models to study DMD are primary X-linked muscular dystrophy (mdx) myoblast”
- Systemic administration of PRO051 in Duchenne's muscular dystrophy. (2011, result): “Inclusion and exclusion criteria were similar to the previous study.(1) An estimated life expectancy of ≥6 months, no other serious pre-existing medical conditions, no assisted ventilation dependency or FEV1 or FVC < 60% of predicted, and no severe muscle abnormalities (defined as increased signal i”
- Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides. (2015, background): “…nity and thus potency of the antisense drug [22 – 24] More recently, it has been demonstrated that antisense drugs can ef fi ciently target muscle tissues,whichhasserved asthebasis for developing therapies for muscular dystrophies such as Duchenne muscular dystrophy and myotonic dystrophy [25 – 27].”