ArticleJournal of human genetics2018
A novel human muscle cell model of Duchenne muscular dystrophy created by CRISPR/Cas9 and evaluation of antisense-mediated exon skipping.
Article in Journal of human genetics, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Who cites it
15 citing papers in PubMed, 35 citations in OpenAlex.
- Antisense oligonucleotide selection scheme for rare Duchenne muscular dystrophy mutations: Application toMolecular therapy. Nucleic acids · 2026Article
- Effect of chemical modification on the exon-skipping activity of heteroduplex oligonucleotides.Molecular therapy. Nucleic acids · 2025Article
- Microdroplet-Engineered Skeletal Muscle Organoids from Primary Tissue Recapitulate Parental Physiology with High Reproducibility.Research (Washington, D.C.) · 2025Article
- Precision in Action: The Role of Clustered Regularly Interspaced Short Palindromic Repeats/Cas in Gene Therapies.Vaccines · 2024Review
- Production of Duchenne muscular dystrophy cellular model using CRISPR-Cas9 exon deletion strategy.Molecular and cellular biochemistry · 2024Article
- Advances in CRISPR/Cas-Based Strategies on Zoonosis.Transboundary and emerging diseases · 2023Review
- Applications of CRISPR-Cas9 as an Advanced Genome Editing System in Life Sciences.Biotech (Basel (Switzerland)) · 2021Review
- Design and In Vitro Evaluation of Splice-Switching Oligonucleotides Bearing Locked Nucleic Acids, Amido-Bridged Nucleic Acids, and Guanidine-Bridged Nucleic Acids.International journal of molecular sciences · 2021Article
- Genome-editing approaches and applications: a brief review on CRISPR technology and its role in cancer.3 Biotech · 2021Review
- Applications of CRISPR/Cas9 in the research of malignant musculoskeletal tumors.BMC musculoskeletal disorders · 2021Review
- Synthetic cannabinoid CP-55,940 induces apoptosis in a human skeletal muscle model via regulation of CBArchives of toxicology · 2021Article
- Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases.Theranostics · 2020Review
- Application whole exome sequencing for the clinical molecular diagnosis of patients with Duchenne muscular dystrophy; identification of four novel nonsense mutations in four unrelated Chinese DMD patients.Molecular genetics & genomic medicine · 2019Article
- Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges.Journal of personalized medicine · 2018Review
- Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy.Journal of personalized medicine · 2018Review
Corrections and comments
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Authors and funding
5 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Oligonucleotide-mediated splicing modulation is a promising therapeutic approach for Duchenne muscular dystrophy (DMD). Recently, eteplirsen, a phosphorodiamidate morpholino oligomer-based splice-switching oligonucleotide (SSO) targeting DMD exon 51, was approved by the U.S. Food and Drug Administration as the first antisense-based drug for DMD patients. For further exploring SSOs targeting other exons in the DMD gene, the efficacy of exon skipping and protein rescue with each SSO sequence needs evaluations in vitro. However, only a few immortalized muscle cell lines derived from DMD patients have been reported and are available to test the efficacy of exon skipping in vitro. To solve this problem, we generated a novel immortalized DMD muscle cell line from the human rhabdomyosarcoma (RD) cell line. We removed DMD exons 51-57 (~0.3 Mb) in the RD cell line using the CRISPR/Cas9 system. Additionally, in this DMD model cell line, we evaluated the exon 50 skipping activity of previously reported SSOs at both the mRNA and protein levels. CRISPR/Cas9-mediated gene editing of the DMD gene in the RD cell line will allow for assessment of SSOs targeting most of the rare mutations in the DMD gene.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.