Cargando…

Saponins enhance exon skipping of 2′-O-methyl phosphorothioate oligonucleotide in vitro and in vivo

BACKGROUND: Antisense oligonucleotide (ASO)-mediated exon skipping has been feasible and promising approach for treating Duchenne muscular dystrophy (DMD) in preclinical and clinical trials, but its therapeutic applications remain challenges due to inefficient delivery. METHODS: We investigated a fe...

Descripción completa

Detalles Bibliográficos
Autores principales:	Wang, Mingxing, Wu, Bo, Shah, Sapana N, Lu, Peijuan, Lu, Qilong
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Dove Medical Press 2018
Materias:	Original Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6217006/ https://www.ncbi.nlm.nih.gov/pubmed/30464402 http://dx.doi.org/10.2147/DDDT.S179008

Ejemplares similares

Polyquaternium-mediated delivery of morpholino oligonucleotides for exon-skipping in vitro and in mdx mice
por: Wang, Mingxing, et al.
Publicado: (2017)

Saponins as Natural Adjuvant for Antisense Morpholino Oligonucleotides Delivery In Vitro and in mdx Mice
por: Wang, Mingxing, 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)

Triazine-cored polymeric vectors for antisense oligonucleotide delivery in vitro and in vivo
por: Wang, Mingxing, et al.
Publicado: (2020)

Tween 85-Modified Low Molecular Weight PEI Enhances Exon-Skipping of Antisense Morpholino Oligomer In Vitro and in mdx Mice
por: Wang, Mingxing, et al.
Publicado: (2017)

Aminoglycoside Enhances the Delivery of Antisense Morpholino Oligonucleotides In Vitro and in mdx Mice
por: Wang, Mingxing, et al.
Publicado: (2019)

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)

An LNA-amide modification that enhances the cell uptake and activity of phosphorothioate exon-skipping oligonucleotides
por: Baker, Ysobel R., et al.
Publicado: (2022)

Evaluation of Amphiphilic Peptide Modified Antisense Morpholino Oligonucleotides In Vitro and in Dystrophic mdx Mice
por: Wang, Mingxing, et al.
Publicado: (2017)

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)

Poly(ester amine) Composed of Polyethylenimine and Pluronic Enhance Delivery of Antisense Oligonucleotides In Vitro and in Dystrophic mdx Mice
por: Wang, Mingxing, et al.
Publicado: (2016)

Rational Design of Short Locked Nucleic Acid-Modified 2′-O-Methyl Antisense Oligonucleotides for Efficient Exon-Skipping In Vitro
por: Le, Bao T., et al.
Publicado: (2017)

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

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

Electronic Structures of LNA Phosphorothioate Oligonucleotides
por: Bohr, Henrik G., et al.
Publicado: (2017)

Phosphorothioate modified oligonucleotide–protein interactions
por: Crooke, Stanley T, et al.
Publicado: (2020)

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

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

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

The exon-skipping oligonucleotide, KitStop, depletes tissue-resident mast cells in vivo to ameliorate anaphylaxis
por: Hedgespeth, Barry A., et al.
Publicado: (2023)

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)

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

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

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)

Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model
por: Heemskerk, Hans, et al.
Publicado: (2010)

Synthesis of a Morpholino Nucleic Acid (MNA)-Uridine Phosphoramidite, and Exon Skipping Using MNA/2′-O-Methyl Mixmer Antisense Oligonucleotide
por: Chen, Suxiang, et al.
Publicado: (2016)

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

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)

Selective Covalent Conjugation of Phosphorothioate DNA Oligonucleotides with Streptavidin
por: Rabe, Kersten S., et al.
Publicado: (2011)

Investigation of factors influencing the separation of diastereomers of phosphorothioated oligonucleotides
por: Enmark, Martin, et al.
Publicado: (2019)

Oligonucleotide Phosphorothioates Enter Cells by Thiol‐Mediated Uptake
por: Laurent, Quentin, et al.
Publicado: (2021)

ExonSkipDB: functional annotation of exon skipping event in human
por: Kim, Pora, et al.
Publicado: (2020)

Fine Tuning of Phosphorothioate Inclusion in 2′-O-Methyl Oligonucleotides Contributes to Specific Cell Targeting for Splice-Switching Modulation
por: Aoki, Yoshitsugu, et al.
Publicado: (2021)

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

Solid-Phase Synthesis of Boranophosphate/Phosphorothioate/Phosphate Chimeric Oligonucleotides and Their Potential as Antisense Oligonucleotides
por: Takahashi, Yuhei, et al.
Publicado: (2021)

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)

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

Cannot write session to /tmp/vufind_sessions/sess_laaprh24dv57b0n9d31pen0c20