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Methods in Molecular Biology (Clifton, N.J.)|November 18, 2022
Developing Therapeutic Splice-Correcting Antisense Oligomers for Adult-Onset Pompe Disease with c.-32-13T>G MutationKristin A Ham, Russell D Johnsen, Michel Tchan, et al.
Immunology and Cell Biology|February 3, 2016
Correcting the NLRP3 inflammasome deficiency in macrophages from autoimmune NZB mice with exon skipping antisense oligonucleotidesSara J Thygesen, David P Sester, Simon O Cridland, et al.
Neuromuscular Disorders : NMD|July 17, 1999
Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotidesS D Wilton, F Lloyd, K Carville, et al.
BMC Molecular Biology|July 3, 2007
Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistriesAbbie M Adams, Penny L Harding, Patrick L Iversen, et al.
Frontiers in Pharmacology|April 8, 2022
Investigating the Implications of CFTR Exon Skipping Using a Cftr Exon 9 Deleted Mouse ModelKelly M Martinovich, Anthony Kicic, Stephen M Stick, et al.
BMC Medical Genetics|October 22, 2011
Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switchingClayton T Fragall, Abbie M Adams, Russell D Johnsen, et al.
Molecular Genetics & Genomic Medicine|February 6, 2014
Antisense suppression of donor splice site mutations in the dystrophin gene transcriptSue Fletcher, Penny L Meloni, Russell D Johnsen, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 22, 2009
Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skippingAurélie Goyenvalle, Arran Babbs, Dave Powell, et al.
The Journal of Gene Medicine|November 15, 2005
Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotideSusan Fletcher, Kaite Honeyman, Abbie M Fall, et al.
Frontiers in Neuroscience|December 24, 2019
ALS Genetics, Mechanisms, and Therapeutics: Where Are We Now?Rita Mejzini, Loren L Flynn, Ianthe L Pitout, et al.
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