In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse.

In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse.

A promising therapeutic approach for Duchenne muscular dystrophy (DMD) is exon skipping using antisense oligonucleotides (AOs). In-frame deletions of the hinge 3 region of the dystrophin protein, which is encoded by exons 50 and 51, are predicted to cause a variety of phenotypes. Here, we performed...

書目詳細資料
Main Authors:	Aoki, Y, Nakamura, A, Yokota, T, Saito, T, Okazawa, H, Nagata, T, Takeda, S
格式:	Journal article
語言:	English
出版:	2010

相似書籍

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.
由: Aoki, Y, et al.
出版: (2012)

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

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

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

A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.
由: Yin, H, et al.
出版: (2009)

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

Good news for the mdx mouse community: Improved dystrophin restoration after skipping mouse dystrophin exon 23
由: Annemieke Aartsma-Rus
出版: (2022-12-01)

Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene
由: Kane L Greer, et al.
出版: (2014-01-01)

Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies.
由: Zhi Yon Charles Toh, et al.
出版: (2016-01-01)

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

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

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

Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment
由: Betts, C, et al.
出版: (2018)

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

Effective exon skipping and dystrophin restoration by 2'-o-methoxyethyl antisense oligonucleotide in dystrophin-deficient mice.
由: Lu Yang, et al.
出版: (2013-01-01)

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

Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene
由: Sue Fletcher, et al.
出版: (2012-01-01)

Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping.
由: Goyenvalle, A, et al.
出版: (2010)

Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping
由: Goyenvalle, A, et al.
出版: (2010)

Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice.
由: Yin, H, et al.
出版: (2011)

Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice
由: Yin, H, et al.
出版: (2011)

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

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

Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice
由: Ngoc Lu-Nguyen, et al.
出版: (2017-03-01)

Exon skipping is correlated with exon circularization
由: Kelly, S, et al.
出版: (2015)

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
由: Shani Attias Cohen, et al.
出版: (2022-09-01)

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

Analysis of Exon Skipping Applicability for Dysferlinopathies
由: Jamie Leckie, et al.
出版: (2025-01-01)

Exon skipping peptide-pmos for correction of dystrophin in mouse models of duchenne muscular dystrophy
由: Betts, C, et al.
出版: (2014)

Context dependent effects of chimeric peptide morpholino conjugates contribute to dystrophin exon-skipping efficiency
由: Yin, HF, et al.
出版: (2016)

Adenine base editing-mediated exon skipping restores dystrophin in humanized Duchenne mouse model
由: Jiajia Lin, et al.
出版: (2024-07-01)

Targeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug development.
由: Bo Wu, et al.
出版: (2011-01-01)

Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency
由: HaiFang Yin, et al.
出版: (2020-12-01)

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

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

Prevention of early-onset cardiomyopathy in Dmd exon 52–54 deletion mice by CRISPR-Cas9-mediated exon skipping
由: Matthew Rok, et al.
出版: (2023-09-01)

Upgrading U7snRNA To Complete Efficient Rescue of Dystrophin by Exon-Skipping in DMD Patients
由: Goyenvalle, A, et al.
出版: (2009)

Identification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screening.
由: Debra A O'Leary, et al.
出版: (2009-12-01)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping
由: Goyenvalle, A, et al.
出版: (2012)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping.
由: Goyenvalle, A, et al.
出版: (2012)