Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Highly sensitive screening of antisense sequences for different types of DMD mutations in patients' urine-derived cells.

Journal of the neurological sciences - 15 Apr 2021

Takizawa Hotake, Takeshita Eri, Sato Mitsuto, Shimizu-Motohashi Yuko, Ishiyama Akihiko, Mori-Yoshimura Madoka, Takahashi Yuji, Komaki Hirofumi, Aoki Yoshitsugu

Abstract excerpt

Exon skipping using short antisense oligonucleotides (AONs) is a promising treatment for Duchenne muscular dystrophy (DMD). Several exon-skipping drugs, including viltolarsen (NS-065/NCNP-01), have been approved worldwide. Immortalized human skeletal muscle cell lines, such as rhabdomyosarcoma cells, are frequently used to screen efficient oligonucleotide sequences. However, rhabdomyosarcoma cells do not...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Dystrophin
Share to Muscular Dystrophy, Duchenne

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Mutation

Share to Oligonucleotides

Share to RNA Splicing

Source

Identifiers

PMID: 33610829
DOI: 10.1016/j.jns.2021.117337

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Highly sensitive screening of antisense sequences for different types of DMD mutations in patients' urine-derived cells.PMID 33610829Read ↗

Quantitative Evaluation of Exon Skipping in Urine-Derived Cells for Duchenne Muscular Dystrophy.Semantic proximity Read ↗

Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy.Semantic proximity Read ↗

Creation of DMD Muscle Cell Model Using CRISPR-Cas9 Genome Editing to Test the Efficacy of Antisense-Mediated Exon Skipping.Semantic proximity Read ↗

Duchenne Muscular Dystrophy Myogenic Cells from Urine-Derived Stem Cells Recapitulate the Dystrophin Genotype and Phenotype.Semantic proximity Read ↗

Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy.Semantic proximity Read ↗

NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular DystrophySemantic proximity Read ↗

Exon skipping: a first in class strategy for Duchenne muscular dystrophySemantic proximity Read ↗

Direct Reprogramming of Human DMD Fibroblasts into Myotubes for In Vitro Evaluation of Antisense-Mediated Exon Skipping and Exons 45-55 Skipping Accompanied by Rescue of Dystrophin Expression.Semantic proximity Read ↗

Recent Trends in Antisense Therapies for Duchenne muscular dystrophySemantic proximity Read ↗

Brogidirsen and Exon 44 Skipping for Duchenne Muscular Dystrophy: Advances and Challenges in RNA-Based TherapySemantic proximity Read ↗

New developments in exon skipping and splice modulation therapies for neuromuscular diseasesSemantic proximity Read ↗

Emerging Oligonucleotide Therapeutics for Rare Neuromuscular DiseasesSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.