Cargando…

Dynamics of Co-Transcriptional Pre-mRNA Folding Influences the Induction of Dystrophin Exon Skipping by Antisense Oligonucleotides

Antisense oligonucleotides (AONs) mediated exon skipping offers potential therapy for Duchenne muscular dystrophy. However, the identification of effective AON target sites remains unsatisfactory for lack of a precise method to predict their binding accessibility. This study demonstrates the importa...

Descripción completa

Detalles Bibliográficos
Autores principales:	Wee, Keng Boon, Pramono, Zacharias Aloysius Dwi, Wang, Jian Li, MacDorman, Karl F., Lai, Poh San, Yee, Woon Chee
Formato:	Texto
Lenguaje:	English
Publicado:	Public Library of Science 2008
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2267000/ https://www.ncbi.nlm.nih.gov/pubmed/18365002 http://dx.doi.org/10.1371/journal.pone.0001844

Ejemplares similares

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

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

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

2′-O-Methyl RNA/Ethylene-Bridged Nucleic Acid Chimera Antisense Oligonucleotides to Induce Dystrophin Exon 45 Skipping
por: Lee, Tomoko, et al.
Publicado: (2017)

Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice
por: Han, Gang, et al.
Publicado: (2016)

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)

Targeted Skipping of Human Dystrophin Exons in Transgenic Mouse Model Systemically for Antisense Drug Development
por: Wu, Bo, et al.
Publicado: (2011)

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

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

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

Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene
por: Greer, Kane L, et al.
Publicado: (2014)

In vivo restoration of dystrophin expression in mdx mice using intra-muscular and intra-arterial injections of hydrogel microsphere carriers of exon skipping antisense oligonucleotides
por: Cohen, Shani Attias, et al.
Publicado: (2022)

Enhanced exon skipping and prolonged dystrophin restoration achieved by TfR1-targeted delivery of antisense oligonucleotide using FORCE conjugation in mdx mice
por: Desjardins, Cody A, et al.
Publicado: (2022)

Nanopolymers improve delivery of exon skipping oligonucleotides and concomitant dystrophin expression in skeletal muscle of mdx mice
por: Williams, Jason H, et al.
Publicado: (2008)

Multiple exon skipping strategies to by-pass dystrophin mutations
por: Adkin, Carl F., et al.
Publicado: (2012)

Elusive sources of variability of dystrophin rescue by exon skipping
por: Vila, Maria Candida, et al.
Publicado: (2015)

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)

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

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)

Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts
por: Lee, Joshua, et al.
Publicado: (2018)

Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene
por: Fletcher, Sue, et al.
Publicado: (2012)

Antisense-Oligonucleotide Mediated Exon Skipping in Activin-Receptor-Like Kinase 2: Inhibiting the Receptor That Is Overactive in Fibrodysplasia Ossificans Progressiva
por: Shi, SongTing, et al.
Publicado: (2013)

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)

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)

Single Exon Skipping Can Address a Multi-Exon Duplication in the Dystrophin Gene
por: Greer, Kane, et al.
Publicado: (2020)

Chemical treatment enhances skipping of a mutated exon in the dystrophin gene
por: Nishida, Atsushi, et al.
Publicado: (2011)

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)

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)

NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy
por: Watanabe, Naoki, et al.
Publicado: (2018)

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)

Cannot write session to /tmp/vufind_sessions/sess_tdtqr9lo5t5p7sprhhq416nqcs