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Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society|July 6, 2021
Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutationYifat S Oren, Michal Irony-Tur Sinai, Anita Golec, et al.Biorxiv : the Preprint Server for Biology|November 21, 2023
Tissue-resident alveolar macrophages reduce O3-induced inflammation via MerTK mediated efferocytosisM A Guttenberg, A T Vose, A Birukova, et al.Frontiers in Aging Neuroscience|April 12, 2021
Novel Variant Linked to Amyotrophic Lateral Sclerosis Risk and Clinical PhenotypeFrances Theunissen, Ryan S Anderton, Frank L Mastaglia, et al.Experimental Eye Research|October 9, 2022
Characterising splicing defects of ABCA4 variants within exons 13-50 in patient-derived fibroblastsDi Huang, Jennifer A Thompson, Shang-Chih Chen, et al.Neuromuscular Disorders : NMD|October 1, 1996
Three novel mutations and two variants in the gene for Cu/Zn superoxide dismutase in familial amyotrophic lateral sclerosisB A Hosler, G A Nicholson, P C Sapp, et al.Journal of Lipid Research|August 3, 2005
Multiple QTLs influencing triglyceride and HDL and total cholesterol levels identified in families with atherogenic dyslipidemiaYi Yu, Diego F Wyszynski, Dawn M Waterworth, et al.Lancet (London, England)|July 26, 2011
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation studySebahattin Cirak, Virginia Arechavala-Gomeza, Michela Guglieri, et al.Proceedings of the National Academy of Sciences of the United States of America|March 3, 1999
Mutations in the nebulin gene associated with autosomal recessive nemaline myopathyK Pelin, P Hilpelä, K Donner, et al.The Lancet. Neurology|August 29, 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 studyMaria Kinali, Virginia Arechavala-Gomeza, Lucy Feng, et al.Nature Medicine|August 11, 2014
Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and miceNicolas Wein, Adeline Vulin, Maria S Falzarano, et al.Pageof 24