Antisense oligonucleotide induced pseudoexon skipping and restoration of functional protein for Fukuyama muscular dystrophy caused by a deep-intronic variant

Biochemistry, Genetics and Molecular Biology

• Antisense 100%
• Oligonucleotide 100%
• Fukutin 83%
• Messenger RNA 33%
• Glycosylation 33%
• Dystroglycan 33%
• Lymphoblast 33%
• Fibroblast 16%
• Nucleotide Sequence 16%
• Enhancer Region 16%
• Untranslated Region 16%
• Intron 16%
• Loss of Function Mutation 16%
• Variable Number Tandem Repeat 16%
• Gene Mutation 16%
• Minigene 16%
• Autosomal Recessive Disorder 16%

Keyphrases

• Splice Correction 14%
• Minigene Splicing Assay 14%
• Oligonucleotide Sequence 14%