Cargando…

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

Duchenne muscular dystrophy (DMD), the most common lethal heritable childhood disease, is caused by mutations in the DMD gene that result in the absence of functional dystrophin protein. Exon skipping mediated by antisense oligonucleotides has recently emerged as an effective approach for the restor...

Descripción completa

Detalles Bibliográficos
Autores principales:	Watanabe, Naoki, Nagata, Tetsuya, Satou, Youhei, Masuda, Satoru, Saito, Takashi, Kitagawa, Hidetoshi, Komaki, Hirofumi, Takagaki, Kazuchika, Takeda, Shin’ichi
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2018
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6202794/ https://www.ncbi.nlm.nih.gov/pubmed/30388618 http://dx.doi.org/10.1016/j.omtn.2018.09.017

Ejemplares similares

Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide
por: Watanabe, Naoki, et al.
Publicado: (2023)

Systemic administration of the antisense oligonucleotide NS‐089/NCNP‐02 for skipping of exon 44 in patients with Duchenne muscular dystrophy: Study protocol for a phase I/II clinical trial
por: Ishizuka, Takami, et al.
Publicado: (2023)

Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy
por: van Deutekom, Judith, et al.
Publicado: (2023)

Evaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy
por: Jirka, Silvana M G, et al.
Publicado: (2015)

Exon-skipping antisense oligonucleotides for cystic fibrosis therapy
por: Kim, Young Jin, et al.
Publicado: (2022)

Wild-Type Mouse Models to Screen Antisense Oligonucleotides for Exon-Skipping Efficacy in Duchenne Muscular Dystrophy
por: Cao, Limin, et al.
Publicado: (2014)

Novel Cationic Carotenoid Lipids as Delivery Vectors of Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy
por: Popplewell, Linda J., et al.
Publicado: (2012)

Antisense Oligonucleotide-Mediated Exon-skipping Therapies: Precision Medicine Spreading from Duchenne Muscular Dystrophy
por: Matsuo, Masafumi
Publicado: (2021)

Antisense Oligonucleotides Conjugated with Lipophilic Compounds: Synthesis and In Vitro Evaluation of Exon Skipping in Duchenne Muscular Dystrophy
por: Marchesi, Elena, et al.
Publicado: (2022)

Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy
por: Aartsma-Rus, Annemieke, et al.
Publicado: (2007)

Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy
por: Jirka, Silvana M.G., et al.
Publicado: (2018)

Correction: Wild-Type Mouse Models to Screen Antisense Oligonucleotides for Exon-Skipping Efficacy in Duchenne Muscular Dystrophy
por: Cao, Limin, et al.
Publicado: (2018)

Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy
por: Aoki, Yoshitsugu, et al.
Publicado: (2013)

Correction: Correction: Wild-Type Mouse Models to Screen Antisense Oligonucleotides for Exon-Skipping Efficacy in Duchenne Muscular Dystrophy
Publicado: (2019)

Digital Droplet PCR for the Absolute Quantification of Exon Skipping Induced by Antisense Oligonucleotides in (Pre-)Clinical Development for Duchenne Muscular Dystrophy
por: Verheul, Ruurd C., et al.
Publicado: (2016)

Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries
por: Adams, Abbie M, et al.
Publicado: (2007)

Proof-of-Concept: Antisense Oligonucleotide Mediated Skipping of Fibrillin-1 Exon 52
por: Cale, Jessica M., et al.
Publicado: (2021)

A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures
por: Hiller, Monika, et al.
Publicado: (2018)

Exon-Skipping in Duchenne Muscular Dystrophy
por: Takeda, Shin’ichi, et al.
Publicado: (2021)

Exon skipping for Duchenne muscular dystrophy: a systematic review and meta-analysis
por: Shimizu-Motohashi, Yuko, et al.
Publicado: (2018)

Cationic polyelectrolyte-mediated delivery of antisense morpholino oligonucleotides for exon-skipping in vitro and in mdx mice
por: Wang, Mingxing, et al.
Publicado: (2015)

Antisense oligonucleotide-mediated exon skipping as a strategy to reduce proteolytic cleavage of ataxin-3
por: Toonen, Lodewijk J. A., et al.
Publicado: (2016)

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy
por: Miskew Nichols, Bailey, et al.
Publicado: (2016)

Systematic evaluation of 2′-Fluoro modified chimeric antisense oligonucleotide-mediated exon skipping in vitro
por: Chen, Suxiang, et al.
Publicado: (2019)

Ensemble-Learning and Feature Selection Techniques for Enhanced Antisense Oligonucleotide Efficacy Prediction in Exon Skipping
por: Zhu, Alex, et al.
Publicado: (2023)

The Effect of 6-Thioguanine on Alternative Splicing and Antisense-Mediated Exon Skipping Treatment for Duchenne Muscular Dystrophy
por: Verhaart, Ingrid E. C., et al.
Publicado: (2012)

Effective Exon Skipping and Dystrophin Restoration by 2′-O-Methoxyethyl Antisense Oligonucleotide in Dystrophin-Deficient Mice
por: Yang, Lu, et al.
Publicado: (2013)

Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides
por: Aartsma-Rus, Annemieke, et al.
Publicado: (2023)

DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon
por: Goossens, Remko, et al.
Publicado: (2023)

eSkip-Finder: a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping
por: Chiba, Shuntaro, et al.
Publicado: (2021)

RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect
por: Domenger, Claire, et al.
Publicado: (2017)

In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy
por: Echigoya, Yusuke, et al.
Publicado: (2015)

Dynamics of Co-Transcriptional Pre-mRNA Folding Influences the Induction of Dystrophin Exon Skipping by Antisense Oligonucleotides
por: Wee, Keng Boon, et al.
Publicado: (2008)

Long-term Exon Skipping Studies With 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models
por: Tanganyika-de Winter, Christa L, et al.
Publicado: (2012)

Author Correction: Systematic evaluation of 2′-Fluoro modified chimeric antisense oligonucleotide-mediated exon skipping in vitro
por: Chen, Suxiang, et al.
Publicado: (2020)

Personalized Development of Antisense Oligonucleotides for Exon Skipping Restores Type XVII Collagen Expression in Junctional Epidermolysis Bullosa
por: Ablinger, Michael, et al.
Publicado: (2021)

Renadirsen, a Novel 2′OMeRNA/ENA(®) Chimera Antisense Oligonucleotide, Induces Robust Exon 45 Skipping for Dystrophin In Vivo
por: Ito, Kentaro, et al.
Publicado: (2021)

Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy
por: Kemaladewi, Dwi U, et al.
Publicado: (2011)

Current Outline of Exon Skipping Trials in Duchenne Muscular Dystrophy
por: Eser, Gökçe, et al.
Publicado: (2022)

Dual Fluorescence Splicing Reporter Minigene Identifies an Antisense Oligonucleotide to Skip Exon v8 of the CD44 Gene
por: Fukushima, Sachiyo, et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_b5rmji423qa79cs4nli5t1i2ct