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Neurodegenerative diseases: a hotbed for splicing defects and the potential therapies
Download from doi.orgProof-of-Concept: Antisense Oligonucleotide Mediated Skipping of Fibrillin-1 Exon 52
Download from doi.orgNovel STMN2 Variant Linked to Amyotrophic Lateral Sclerosis Risk and Clinical Phenotype
Download from doi.orgNonsequential Splicing Events Alter Antisense-Mediated Exon Skipping Outcome in COL7A1
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