A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.

A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.

Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene that abolish the synthesis of dystrophin protein. Antisense oligonucleotides (AOs) targeted to trigger excision of an exon bearing a mutant premature stop codon in the DMD transcript have been shown to skip the mutated exon and...

書誌詳細
主要な著者:	Yin, H, Moulton, H, Betts, C, Seow, Y, Boutilier, J, Iverson, P, Wood, M
フォーマット:	Journal article
言語:	English
出版事項:	2009

類似資料

Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO
著者:: Yin, H, 等
出版事項: (2010)

Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice
著者:: Gang Han, 等
出版事項: (2016-03-01)

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

Context dependent effects of chimeric peptide morpholino conjugates contribute to dystrophin exon-skipping efficiency
著者:: Yin, HF, 等
出版事項: (2016)

Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment
著者:: Betts, C, 等
出版事項: (2018)

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

Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice
著者:: Yin, H, 等
出版事項: (2011)

Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency
著者:: HaiFang Yin, 等
出版事項: (2020-12-01)

Effective exon skipping and dystrophin restoration by 2'-o-methoxyethyl antisense oligonucleotide in dystrophin-deficient mice.
著者:: Lu Yang, 等
出版事項: (2013-01-01)

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse.
著者:: Lu, Q, 等
出版事項: (2003)

In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse.
著者:: Aoki, Y, 等
出版事項: (2010)

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

Use of antisense-mediated exon skipping to generate dystrophin in muscles of the mdx dystrophic mouse
著者:: Lu, Q, 等
出版事項: (2004)

Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function.
著者:: Yin, H, 等
出版事項: (2008)

Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice
著者:: Xianjun Gao, 等
出版事項: (2015-01-01)

Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-Deficient mdx Mice
著者:: Xianjun Gao, 等
出版事項: (2020-12-01)

Exon skipping peptide-pmos for correction of dystrophin in mouse models of duchenne muscular dystrophy
著者:: Betts, C, 等
出版事項: (2014)

Increased neointimal thickening in dystrophin-deficient mdx mice.
著者:: Uwe Rauch, 等
出版事項: (2012-01-01)

Rescue of a dystrophin-like protein by exon skipping normalizes synaptic plasticity in the hippocampus of the mdx mouse
著者:: Glenn Dallérac, 等
出版事項: (2011-09-01)

Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene
著者:: Kane L Greer, 等
出版事項: (2014-01-01)

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
著者:: Shani Attias Cohen, 等
出版事項: (2022-09-01)

Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping
著者:: Goyenvalle, A, 等
出版事項: (2010)

Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse.
著者:: Yin, H, 等
出版事項: (2010)

Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene
著者:: Sue Fletcher, 等
出版事項: (2012-01-01)

Adenine base editing-mediated exon skipping restores dystrophin in humanized Duchenne mouse model
著者:: Jiajia Lin, 等
出版事項: (2024-07-01)

Muscle regeneration in dystrophin-deficient <it>mdx </it>mice studied by gene expression profiling
著者:: van Ommen G-JB, 等
出版事項: (2005-07-01)

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

The proton pump inhibitor lansoprazole improves the skeletal phenotype in dystrophin deficient mdx mice.
著者:: Arpana Sali, 等
出版事項: (2013-01-01)

Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.
著者:: Yokota, T, 等
出版事項: (2012)

Effect of 2-Aminoethoxydiphenyl Borate on the State of Skeletal Muscles in Dystrophin-Deficient mdx Mice
著者:: Mikhail V. Dubinin, 等
出版事項: (2024-12-01)

Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice
著者:: Ngoc Lu-Nguyen, 等
出版事項: (2017-03-01)

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

Microarray analysis of mdx mice expressing high levels of utrophin: therapeutic implications for dystrophin deficiency.
著者:: Baban, D, 等
出版事項: (2008)

Mitochondrial Dysfunction Is an Early Consequence of Partial or Complete Dystrophin Loss in mdx Mice
著者:: Timothy M. Moore, 等
出版事項: (2020-06-01)

Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties
著者:: Chwalenia, K, 等
出版事項: (2022)

Testing of SHIRPA, a mouse phenotypic assessment protocol, on Dmd(mdx) and Dmd(mdx3cv) dystrophin-deficient mice.
著者:: Rafael, J, 等
出版事項: (2000)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping
著者:: Goyenvalle, A, 等
出版事項: (2012)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping.
著者:: Goyenvalle, A, 等
出版事項: (2012)

Metabolic remodeling agents show beneficial effects in the dystrophin<it>-</it>deficient <it>mdx</it> mouse model
著者:: Jahnke Vanessa E, 等
出版事項: (2012-08-01)

Correction: Functional and Molecular Effects of Arginine Butyrate and Prednisone on Muscle and Heart in the Dystrophin Deficient mdx mice
出版事項: (2010-01-01)