Cargando…

DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon

Mutations in the DMD gene are causative for Duchenne muscular dystrophy (DMD). Antisense oligonucleotide (AON) mediated exon skipping to restore disrupted dystrophin reading frame is a therapeutic approach that allows production of a shorter but functional protein. As DMD causing mutations can affec...

Descripción completa

Detalles Bibliográficos
Autores principales:	Goossens, Remko, Verwey, Nisha, Ariyurek, Yavuz, Schnell, Fred, Aartsma-Rus, Annemieke
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Taylor & Francis 2023
Materias:	Research Paper
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10481881/ https://www.ncbi.nlm.nih.gov/pubmed/37667454 http://dx.doi.org/10.1080/15476286.2023.2254041

Ejemplares similares

Exon skipping for DMD
por: Aartsma-Rus, Annemieke, et al.
Publicado: (2012)

Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice
por: Bizot, Flavien, et al.
Publicado: (2022)

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

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)

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

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)

Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy
por: van Deutekom, Judith, 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)

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)

Nonclinical Exon Skipping Studies with 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn−/− Mice Inspired by Clinical Trial Results
por: van Putten, Maaike, et al.
Publicado: (2019)

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

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)

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)

Therapeutic exon skipping for dysferlinopathies?
por: Aartsma-Rus, Annemieke, et al.
Publicado: (2010)

Good news for the mdx mouse community: Improved dystrophin restoration after skipping mouse dystrophin exon 23
por: Aartsma-Rus, Annemieke
Publicado: (2022)

Inhibition of IL-1 Signaling by Antisense Oligonucleotide-mediated Exon Skipping of IL-1 Receptor Accessory Protein (IL-1RAcP)
por: Yılmaz-Eliş, A Seda, et al.
Publicado: (2013)

Skipping Multiple Exons to Treat DMD—Promises and Challenges
por: Aslesh, Tejal, et al.
Publicado: (2018)

Shorter Phosphorodiamidate Morpholino Splice-Switching Oligonucleotides May Increase Exon-Skipping Efficacy in DMD
por: Akpulat, Ugur, et al.
Publicado: (2018)

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)

Prime editing strategies to mediate exon skipping in DMD gene
por: Happi Mbakam, Cedric, et al.
Publicado: (2023)

Repurposing Pathogenic Variants of DMD Gene and its Isoforms for DMD Exon Skipping Intervention
por: Tyagi, Rahul, et al.
Publicado: (2019)

Novel Exon-Skipping Therapeutic Approach for the DMD Gene Based on Asymptomatic Deletions of Exon 49
por: Abaji, Mario, et al.
Publicado: (2022)

Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy
por: Gramlich, Michael, et al.
Publicado: (2015)

Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse
por: Wang, Derek W., et al.
Publicado: (2018)

Endogenous Multiple Exon Skipping and Back-Splicing at the DMD Mutation Hotspot
por: Suzuki, Hitoshi, et al.
Publicado: (2016)

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)

Assessment of the feasibility of exon 45–55 multiexon skipping for duchenne muscular dystrophy
por: van Vliet, Laura, et al.
Publicado: (2008)

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

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

What Can We Learn From Clinical Trials of Exon Skipping for DMD?
por: Lu, Qi-long, et al.
Publicado: (2014)

Targeting Duchenne muscular dystrophy by skipping DMD exon 45 with base editors
por: Gapinske, Michael, et al.
Publicado: (2023)

Effective Exon Skipping and Dystrophin Restoration by 2′-O-Methoxyethyl Antisense Oligonucleotide in Dystrophin-Deficient Mice
por: Yang, Lu, 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)

Cannot write session to /tmp/vufind_sessions/sess_s87bu26qbkbce6ju4i3m390sad