Results 171 to 180 of about 29,161 (207)

The association of nonsense codons with exon skipping

Mutation Research/Reviews in Mutation Research, 1998
Some genes that contain premature nonsense codons express alternatively-spliced mRNA that has skipped the exon containing the nonsense codon. This paradoxical association of translation signals (nonsense codons) and RNA splicing has inspired numerous explanations. The first is based on the fact that premature nonsense codons often reduce mRNA abundance.
openaire   +2 more sources

Overview on Applications of Antisense-Mediated Exon Skipping

2012
Antisense-mediated exon skipping has multiple therapeutic applications. This chapter gives an overview of how this tool has been employed to restore normal splicing for cryptic splicing mutations, to switch between alternative splicing isoforms, to induce exon inclusion, to correct the reading frame to allow the production of internally deleted ...
Willeke M C, van Roon-Mom, Annemieke, Aartsma-Rus   +1 more
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Overview of Alternative Oligonucleotide Chemistries for Exon Skipping

2012
The chemistry of the oligonucleotide backbone is crucial to obtaining high activity in vivo in exon skipping applications. Apart from the ability to bind strongly and sequence-specifically to pre-mRNA targets, the type of backbone also influences cell delivery, in vivo pharmacology, bio-distribution, toxicology, and ultimately the therapeutic use in ...
Amer F, Saleh, Andrey A, Arzumanov, Michael J, Gait   +2 more
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Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy

Proceedings of the National Academy of Sciences of the United States of America, 2022
Md Nur Ahad Shāh, Toshifumi Yokota, Stanley Woo   +2 more
exaly  

Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide

Molecular Therapy - Nucleic Acids, 2023
Tetsuya Nagata, Norio Motohashi, Yoshitsugu Aoki   +2 more
exaly  

The promise of selective MET inhibitors in non-small cell lung cancer with MET exon 14 skipping

Cancer Treatment Reviews, 2020
Martin Sattler, Ravi Salgia, Eudald Felip   +2 more
exaly  

Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy

Nucleic Acid Therapeutics, 2023
Annemieke Aartsma-Rus, Jukka Puoliväli   +1 more
exaly  

Current Outline of Exon Skipping Trials in Duchenne Muscular Dystrophy

Genes, 2022
Gökçe Eser, Halûk Topaloğlu
exaly  

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

Human Mutation, 2009
Jan Verschuuren, Johan T Den Dunnen, Annemieke Aartsma-Rus   +2 more
exaly  

Exon skipping therapy for DMD

Journal of Neuromuscular Diseases, 2021
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