Cargando…

Nanopolymers improve delivery of exon skipping oligonucleotides and concomitant dystrophin expression in skeletal muscle of mdx mice

BACKGROUND: Exon skipping oligonucleotides (ESOs) of 2'O-Methyl (2'OMe) and morpholino chemistry have been shown to restore dystrophin expression in muscle fibers from the mdx mouse, and are currently being tested in phase I clinical trials for Duchenne Muscular Dystrophy (DMD). However, E...

Descripción completa

Detalles Bibliográficos
Autores principales:	Williams, Jason H, Schray, Rebecca C, Sirsi, Shashank R, Lutz, Gordon J
Formato:	Texto
Lenguaje:	English
Publicado:	BioMed Central 2008
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2362111/ https://www.ncbi.nlm.nih.gov/pubmed/18384691 http://dx.doi.org/10.1186/1472-6750-8-35

Ejemplares similares

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

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

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

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

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

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

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)

Partial Restoration of Brain Dystrophin and Behavioral Deficits by Exon Skipping in the Muscular Dystrophy X‐Linked (mdx) Mouse
por: Zarrouki, Faouzi, et al.
Publicado: (2022)

Formulation of polylactide-co-glycolic acid nanospheres for encapsulation and sustained release of poly(ethylene imine)-poly(ethylene glycol) copolymers complexed to oligonucleotides
por: Sirsi, Shashank R, et al.
Publicado: (2009)

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

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

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)

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)

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)

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)

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

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

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)

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

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)

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

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

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)

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

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)

Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells
por: Lehto, Taavi, et al.
Publicado: (2014)

Interrogation of Dystrophin and Dystroglycan Complex Protein Turnover After Exon Skipping Therapy
por: Novak, James S., et al.
Publicado: (2021)

Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies
por: Toh, Zhi Yon Charles, et al.
Publicado: (2016)

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

A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping
por: Echigoya, Yusuke, et al.
Publicado: (2021)

Evaluation of exon-skipping strategies for Duchenne muscular dystrophy utilizing dystrophin-deficient zebrafish
por: Berger, Joachim, et al.
Publicado: (2011)

Dystrophin-associated proteins are greatly reduced in skeletal muscle from mdx mice
Publicado: (1991)

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

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

Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches
por: Lorain, Stéphanie, 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)

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)

Effect of rapamycin on immunity induced by vector-mediated dystrophin expression in mdx skeletal muscle
por: Eghtesad, Saman, et al.
Publicado: (2012)

Cannot write session to /tmp/vufind_sessions/sess_cpp9ms4o4ga79vp85uklmnonu1