Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

Duchenne muscular dystrophy (DMD), the commonest form of muscular dystrophy, is caused by lack of dystrophin. One of the most promising therapeutic approaches is antisense-mediated elimination of frame-disrupting mutations by exon skipping. However, this approach faces two major hurdles: limited app...

Full description

Bibliographic Details
Main Authors:	Aoki, Y, Yokota, T, Nagata, T, Nakamura, A, Tanihata, J, Saito, T, Duguez, S, Nagaraju, K, Hoffman, E, Partridge, T, Takeda, S
Format:	Journal article
Language:	English
Published:	2012

Similar Items

Use of antisense-mediated exon skipping to generate dystrophin in muscles of the mdx dystrophic mouse
by: Lu, Q, et al.
Published: (2004)

Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice
by: Yusuke Echigoya, et al.
Published: (2015-01-01)

In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse.
by: Aoki, Y, et al.
Published: (2010)

Mutation types and aging differently affect revertant fiber expansion in dystrophic mdx and mdx52 mice.
by: Yusuke Echigoya, et al.
Published: (2013-01-01)

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse.
by: Lu, Q, et al.
Published: (2003)

Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.
by: Yokota, T, et al.
Published: (2012)

New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy.
by: Aoki, Y, et al.
Published: (2012)

Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice
by: Flavien Bizot, et al.
Published: (2022-12-01)

Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts.
by: Joshua Lee, et al.
Published: (2018-01-01)

Challenges for antisense oligonucleotide-based therapeutics, in particular for exon 51-skipping in Duchenne muscular dystrophy
by: Aoki, Y, et al.
Published: (2011)

Proof-of-Concept: Antisense Oligonucleotide Mediated Skipping of Fibrillin-1 Exon 52
by: Jessica M. Cale, et al.
Published: (2021-03-01)

Cationic polyelectrolyte-mediated delivery of antisense morpholino oligonucleotides for exon-skipping in vitro and in mdx mice
by: Wang M, et al.
Published: (2015-09-01)

Natural Exon Skipping Sets the Stage for Exon Skipping as Therapy for Dystrophic Epidermolysis Bullosa
by: Jeroen Bremer, et al.
Published: (2019-12-01)

Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient
by: Saito, T, et al.
Published: (2010)

Antisense Oligonucleotide-mediated Exon Skipping as a Systemic Therapeutic Approach for Recessive Dystrophic Epidermolysis Bullosa
by: Jeroen Bremer, et al.
Published: (2016-01-01)

Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse
by: Derek W. Wang, et al.
Published: (2018-06-01)

Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient.
by: Takashi Saito, et al.
Published: (2010-01-01)

Networking to Optimize <i>Dmd</i> exon 53 Skipping in the Brain of <i>mdx52</i> Mouse Model
by: Mathilde Doisy, et al.
Published: (2023-12-01)

Tween 85-Modified Low Molecular Weight PEI Enhances Exon-Skipping of Antisense Morpholino Oligomer In Vitro and in mdx Mice
by: Mingxing Wang, et al.
Published: (2017-12-01)

Ensemble-Learning and Feature Selection Techniques for Enhanced Antisense Oligonucleotide Efficacy Prediction in Exon Skipping
by: Alex Zhu, et al.
Published: (2023-06-01)

Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice
by: Philippine Aupy, et al.
Published: (2020-06-01)

A <i>Dystrophin</i> Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping
by: Yusuke Echigoya, et al.
Published: (2021-12-01)

T-cell-dependent fibrosis in the mdx dystrophic mouse.
by: Morrison, J, et al.
Published: (2000)

Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice
by: Gang Han, et al.
Published: (2016-03-01)

Identification of Novel Antisense-Mediated Exon Skipping Targets in DYSF for Therapeutic Treatment of Dysferlinopathy
by: Joshua J.A. Lee, et al.
Published: (2018-12-01)

Efficient Modulation of Exon Skipping via Antisense Circular RNAs
by: Shuaiwei Ren, et al.
Published: (2023-01-01)

Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy
by: van Ommen Gert-Jan B, et al.
Published: (2007-07-01)

Expansion of revertant fibers in dystrophic mdx muscles reflects activity of muscle precursor cells and serves as an index of muscle regeneration.
by: Yokota, T, et al.
Published: (2006)

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
by: Shani Attias Cohen, et al.
Published: (2022-09-01)

NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy
by: Naoki Watanabe, et al.
Published: (2018-12-01)

Intramuscular Evaluation of Chimeric Locked Nucleic Acid/2′<i>O</i>Methyl-Modified Antisense Oligonucleotides for Targeted Exon 23 Skipping in Mdx Mice
by: Michaella Georgiadou, et al.
Published: (2021-10-01)

Poly(ester amine) Composed of Polyethylenimine and Pluronic Enhance Delivery of Antisense Oligonucleotides In Vitro and in Dystrophic mdx Mice
by: Mingxing Wang, et al.
Published: (2016-01-01)

Oligonucleotide Enhancing Compound Increases Tricyclo-DNA Mediated Exon-Skipping Efficacy in the Mdx Mouse Model
by: Flavien Bizot, et al.
Published: (2023-02-01)

Nanopolymers improve delivery of exon skipping oligonucleotides and concomitant dystrophin expression in skeletal muscle of <it>mdx </it>mice
by: Sirsi Shashank R, et al.
Published: (2008-04-01)

Rescue of a dystrophin-like protein by exon skipping normalizes synaptic plasticity in the hippocampus of the mdx mouse
by: Glenn Dallérac, et al.
Published: (2011-09-01)

Heregulin ameliorates the dystrophic phenotype in mdx mice.
by: Krag, T, et al.
Published: (2004)

Decrease in prosaposin in the Dystrophic mdx mouse brain.
by: Hui-Ling Gao, et al.
Published: (2013-01-01)

Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy
by: Blain, A, et al.
Published: (2018)

Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy.
by: Alison M Blain, et al.
Published: (2018-01-01)

Antisense‐mediated exon skipping: a therapeutic strategy for titin‐based dilated cardiomyopathy
by: Michael Gramlich, et al.
Published: (2015-05-01)