Cargando…

Nonclinical Exon Skipping Studies with 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn−/− Mice Inspired by Clinical Trial Results

Duchenne muscular dystrophy is a severe, progressive muscle-wasting disease that is caused by mutations that abolish the production of functional dystrophin protein. The exon skipping approach aims to restore the disrupted dystrophin reading frame, to allow the production of partially functional dys...

Descripción completa

Detalles Bibliográficos
Autores principales:	van Putten, Maaike, Tanganyika-de Winter, Christa, Bosgra, Sieto, Aartsma-Rus, Annemieke
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Mary Ann Liebert, Inc., publishers 2019
Materias:	Original Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6461150/ https://www.ncbi.nlm.nih.gov/pubmed/30672725 http://dx.doi.org/10.1089/nat.2018.0759

Ejemplares similares

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)

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

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)

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

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

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)

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)

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)

A comparison of the bone and growth phenotype of mdx, mdx:Cmah(−/−) and mdx:Utrn(+/−) murine models with the C57BL/10 wild-type mouse
por: Wood, Claire L., 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)

Assessment of Behavioral Characteristics With Procedures of Minimal Human Interference in the mdx Mouse Model for Duchenne Muscular Dystrophy
por: Engelbeen, Sarah, et al.
Publicado: (2021)

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)

The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice
por: Verhaart, Ingrid E C, et al.
Publicado: (2014)

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)

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

Natural disease history of the D2-mdx mouse model for Duchenne muscular dystrophy
por: van Putten, Maaike, et al.
Publicado: (2019)

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

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)

Induction of revertant fibres in the mdx mouse using antisense oligonucleotides
por: Fall, Abbie M, et al.
Publicado: (2006)

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)

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)

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)

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

Plasma lipidomic analysis shows a disease progression signature in mdx mice
por: Tsonaka, Roula, et al.
Publicado: (2021)

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)

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

Detailed genetic and functional analysis of the hDMDdel52/mdx mouse model
por: Yavas, Alper, et al.
Publicado: (2020)

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

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)

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)

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

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)

Skeletal Muscle Fibrosis in the mdx/utrn+/- Mouse Validates Its Suitability as a Murine Model of Duchenne Muscular Dystrophy
por: Gutpell, Kelly M., et al.
Publicado: (2015)

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

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)

Cannot write session to /tmp/vufind_sessions/sess_2qsqp35ihl3flcjkhnb8rll737