Cargando…

Polyquaternium-mediated delivery of morpholino oligonucleotides for exon-skipping in vitro and in mdx mice

Antisense oligonucleotide therapy for Duchenne muscular dystrophy has shown great potential in preclinical and clinical trials, but its therapeutic applications are still limited due to inefficient delivery. In this study, we investigated a few polyquaterniums (PQs) with different size and compositi...

Descripción completa

Detalles Bibliográficos
Autores principales:	Wang, Mingxing, Wu, Bo, Shah, Sapana N, Lu, Peijuan, Lu, Qilong
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Taylor & Francis 2017
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8241187/ https://www.ncbi.nlm.nih.gov/pubmed/28633548 http://dx.doi.org/10.1080/10717544.2017.1337827

Ejemplares similares

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

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

Saponins as Natural Adjuvant for Antisense Morpholino Oligonucleotides Delivery In Vitro and in mdx Mice
por: Wang, Mingxing, et al.
Publicado: (2018)

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)

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

Saponins enhance exon skipping of 2′-O-methyl phosphorothioate oligonucleotide in vitro and in vivo
por: Wang, Mingxing, et al.
Publicado: (2018)

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)

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

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

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)

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

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)

Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice
por: Echigoya, Yusuke, et al.
Publicado: (2015)

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)

Oligonucleotide Enhancing Compound Increases Tricyclo-DNA Mediated Exon-Skipping Efficacy in the Mdx Mouse Model
por: Bizot, Flavien, et al.
Publicado: (2023)

Using a State-of-the-Art Toolbox to Evaluate Molecular and Functional Readouts of Antisense Oligonucleotide-Induced Exon Skipping in mdx Mice
por: Datson, Nicole A., et al.
Publicado: (2020)

Intramuscular Evaluation of Chimeric Locked Nucleic Acid/2′OMethyl-Modified Antisense Oligonucleotides for Targeted Exon 23 Skipping in Mdx Mice
por: Georgiadou, Michaella, et al.
Publicado: (2021)

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

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)

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)

A cell-penetrating peptide enhances delivery and efficacy of phosphorodiamidate morpholino oligomers in mdx mice
por: Gan, Li, et al.
Publicado: (2022)

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

Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency
por: Yin, HaiFang, et al.
Publicado: (2013)

Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency
por: Yin, HaiFang, et al.
Publicado: (2020)

The use of urinary and kidney SILAM proteomics to monitor kidney response to high dose morpholino oligonucleotides in the mdx mouse
por: Zhang, Aiping, et al.
Publicado: (2015)

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

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)

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)

Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment
por: Betts, Corinne A, et al.
Publicado: (2019)

Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice
por: Aupy, Philippine, et al.
Publicado: (2020)

Peptide Nanoparticle Delivery of Charge-Neutral Splice-Switching Morpholino Oligonucleotides
por: Järver, Peter, et al.
Publicado: (2015)

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)

Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy
por: Blain, Alison M., 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)

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

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)

Long-Term Morpholino Oligomers in Hexose Elicits Long-Lasting Therapeutic Improvements in mdx Mice
por: Han, Gang, et al.
Publicado: (2018)

Cannot write session to /tmp/vufind_sessions/sess_uuq8rdga0f7htu98ugg3jbt24g