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...

Bibliografski detalji
Glavni autori:	Aoki, Y, Nakamura, A, Yokota, T, Saito, T, Okazawa, H, Nagata, T, Takeda, S
Format:	Journal article
Jezik:	English
Izdano:	2010

Slični predmeti

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.
od: Aoki, Y, i dr.
Izdano: (2012)

Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice
od: Gang Han, i dr.
Izdano: (2016-03-01)

Use of antisense-mediated exon skipping to generate dystrophin in muscles of the mdx dystrophic mouse
od: Lu, Q, i dr.
Izdano: (2004)

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse.
od: Lu, Q, i dr.
Izdano: (2003)

A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.
od: Yin, H, i dr.
Izdano: (2009)

Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.
od: Yokota, T, i dr.
Izdano: (2012)

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

Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene
od: Kane L Greer, i dr.
Izdano: (2014-01-01)

Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies.
od: Zhi Yon Charles Toh, i dr.
Izdano: (2016-01-01)

Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice
od: Philippine Aupy, i dr.
Izdano: (2020-06-01)

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

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

Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment
od: Betts, C, i dr.
Izdano: (2018)

Challenges for antisense oligonucleotide-based therapeutics, in particular for exon 51-skipping in Duchenne muscular dystrophy
od: Aoki, Y, i dr.
Izdano: (2011)

Effective exon skipping and dystrophin restoration by 2'-o-methoxyethyl antisense oligonucleotide in dystrophin-deficient mice.
od: Lu Yang, i dr.
Izdano: (2013-01-01)

Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts.
od: Joshua Lee, i dr.
Izdano: (2018-01-01)

Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene
od: Sue Fletcher, i dr.
Izdano: (2012-01-01)

Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping.
od: Goyenvalle, A, i dr.
Izdano: (2010)

Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping
od: Goyenvalle, A, i dr.
Izdano: (2010)

Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice.
od: Yin, H, i dr.
Izdano: (2011)

Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice
od: Yin, H, i dr.
Izdano: (2011)

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

New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy.
od: Aoki, Y, i dr.
Izdano: (2012)

Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice
od: Ngoc Lu-Nguyen, i dr.
Izdano: (2017-03-01)

Exon skipping is correlated with exon circularization
od: Kelly, S, i dr.
Izdano: (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
od: Shani Attias Cohen, i dr.
Izdano: (2022-09-01)

Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice
od: Flavien Bizot, i dr.
Izdano: (2022-12-01)

Analysis of Exon Skipping Applicability for Dysferlinopathies
od: Jamie Leckie, i dr.
Izdano: (2025-01-01)

Exon skipping peptide-pmos for correction of dystrophin in mouse models of duchenne muscular dystrophy
od: Betts, C, i dr.
Izdano: (2014)

Context dependent effects of chimeric peptide morpholino conjugates contribute to dystrophin exon-skipping efficiency
od: Yin, HF, i dr.
Izdano: (2016)

Adenine base editing-mediated exon skipping restores dystrophin in humanized Duchenne mouse model
od: Jiajia Lin, i dr.
Izdano: (2024-07-01)

Targeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug development.
od: Bo Wu, i dr.
Izdano: (2011-01-01)

Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency
od: HaiFang Yin, i dr.
Izdano: (2020-12-01)

Oligonucleotide Enhancing Compound Increases Tricyclo-DNA Mediated Exon-Skipping Efficacy in the Mdx Mouse Model
od: Flavien Bizot, i dr.
Izdano: (2023-02-01)

Cationic polyelectrolyte-mediated delivery of antisense morpholino oligonucleotides for exon-skipping in vitro and in mdx mice
od: Wang M, i dr.
Izdano: (2015-09-01)

Prevention of early-onset cardiomyopathy in Dmd exon 52–54 deletion mice by CRISPR-Cas9-mediated exon skipping
od: Matthew Rok, i dr.
Izdano: (2023-09-01)

Upgrading U7snRNA To Complete Efficient Rescue of Dystrophin by Exon-Skipping in DMD Patients
od: Goyenvalle, A, i dr.
Izdano: (2009)

Identification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screening.
od: Debra A O'Leary, i dr.
Izdano: (2009-12-01)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping
od: Goyenvalle, A, i dr.
Izdano: (2012)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping.
od: Goyenvalle, A, i dr.
Izdano: (2012)