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Exon skipping in Duchenne muscular dystrophy
Neuromuscular Disorders, 2008What the journals ...
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Designing Effective Antisense Oligonucleotides for Exon Skipping
2017During the past 10Â years, antisense oligonucleotide-mediated exon skipping and splice modulation have proven to be powerful tools for correction of mRNA splicing in genetic diseases. In 2016, the US Food and Drug Administration (FDA)-approved Exondys 51 (eteplirsen) and Spinraza (nusinersen), the first exon skipping and exon inclusion drugs, to treat ...
Takenori Shimo +4 more
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Pharmacology and toxicology of eteplirsen and SRP-5051 for DMD exon 51 skipping: an update
Archives of Toxicology, 2021O. Sheikh, T. Yokota
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Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy
Gene Therapy, 2020K. Dzierlega, T. Yokota
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Exon-Skipping–Based Subtyping of Colorectal Cancers
GastroenterologyThe identification of colorectal cancer (CRC) molecular subtypes has prognostic and potentially diagnostic value for patients, yet reliable subtyping remains unavailable in the clinic. The current consensus molecular subtype (CMS) classification in CRCs is based on complex RNA expression patterns quantified at the gene level.
Aslihan Ambeskovic +4 more
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Management of Non-small Cell Lung Cancer Patients with MET Exon 14 Skipping Mutations
Current Treatment Options in Oncology, 2020Caiwen Huang +6 more
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A novel exonic mutation in the aspartylglucosaminidase gene causes exon skipping
Journal of Inherited Metabolic Disease, 1999openaire +3 more sources