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6. Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs. Yokota T; Nakamura A; Nagata T; Saito T; Kobayashi M; Aoki Y; Echigoya Y; Partridge T; Hoffman EP; Takeda S Nucleic Acid Ther; 2012 Oct; 22(5):306-15. PubMed ID: 22888777 [TBL] [Abstract][Full Text] [Related]
7. Causes of clinical variability in Duchenne and Becker muscular dystrophies and implications for exon skipping therapies. Hoffman EP Acta Myol; 2020 Dec; 39(4):179-186. PubMed ID: 33458572 [TBL] [Abstract][Full Text] [Related]
8. Biochemical characterization of patients with in-frame or out-of-frame DMD deletions pertinent to exon 44 or 45 skipping. Anthony K; Arechavala-Gomeza V; Ricotti V; Torelli S; Feng L; Janghra N; Tasca G; Guglieri M; Barresi R; Armaroli A; Ferlini A; Bushby K; Straub V; Ricci E; Sewry C; Morgan J; Muntoni F JAMA Neurol; 2014 Jan; 71(1):32-40. PubMed ID: 24217213 [TBL] [Abstract][Full Text] [Related]
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10. DMD transcript imbalance determines dystrophin levels. Spitali P; van den Bergen JC; Verhaart IE; Wokke B; Janson AA; van den Eijnde R; den Dunnen JT; Laros JF; Verschuuren JJ; 't Hoen PA; Aartsma-Rus A FASEB J; 2013 Dec; 27(12):4909-16. PubMed ID: 23975932 [TBL] [Abstract][Full Text] [Related]
11. A comparative study of N-glycolylneuraminic acid (Neu5Gc) and cytotoxic T cell (CT) carbohydrate expression in normal and dystrophin-deficient dog and human skeletal muscle. Martin PT; Golden B; Okerblom J; Camboni M; Chandrasekharan K; Xu R; Varki A; Flanigan KM; Kornegay JN PLoS One; 2014; 9(2):e88226. PubMed ID: 24505439 [TBL] [Abstract][Full Text] [Related]
12. Pharmacological rescue of the dystrophin-glycoprotein complex in Duchenne and Becker skeletal muscle explants by proteasome inhibitor treatment. Assereto S; Stringara S; Sotgia F; Bonuccelli G; Broccolini A; Pedemonte M; Traverso M; Biancheri R; Zara F; Bruno C; Lisanti MP; Minetti C Am J Physiol Cell Physiol; 2006 Feb; 290(2):C577-82. PubMed ID: 16192300 [TBL] [Abstract][Full Text] [Related]
13. Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies. Toh ZY; Thandar Aung-Htut M; Pinniger G; Adams AM; Krishnaswarmy S; Wong BL; Fletcher S; Wilton SD PLoS One; 2016; 11(1):e0145620. PubMed ID: 26745801 [TBL] [Abstract][Full Text] [Related]
14. A dystrophic Duchenne mouse model for testing human antisense oligonucleotides. Veltrop M; van Vliet L; Hulsker M; Claassens J; Brouwers C; Breukel C; van der Kaa J; Linssen MM; den Dunnen JT; Verbeek S; Aartsma-Rus A; van Putten M PLoS One; 2018; 13(2):e0193289. PubMed ID: 29466448 [TBL] [Abstract][Full Text] [Related]
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16. Alterations in Notch signalling in skeletal muscles from mdx and dko dystrophic mice and patients with Duchenne muscular dystrophy. Church JE; Trieu J; Chee A; Naim T; Gehrig SM; Lamon S; Angelini C; Russell AP; Lynch GS Exp Physiol; 2014 Apr; 99(4):675-87. PubMed ID: 24443351 [TBL] [Abstract][Full Text] [Related]
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19. In Vivo Evaluation of Multiple Exon Skipping with Peptide-PMOs in Cardiac and Skeletal Muscles in Dystrophic Dogs. Maruyama R; Aoki Y; Takeda S; Yokota T Methods Mol Biol; 2018; 1828():365-379. PubMed ID: 30171554 [TBL] [Abstract][Full Text] [Related]
20. Temporal gene expression profiling of dystrophin-deficient (mdx) mouse diaphragm identifies conserved and muscle group-specific mechanisms in the pathogenesis of muscular dystrophy. Porter JD; Merriam AP; Leahy P; Gong B; Feuerman J; Cheng G; Khanna S Hum Mol Genet; 2004 Feb; 13(3):257-69. PubMed ID: 14681298 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]