Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD

Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD

<div>Duchenne muscular dystrophy (DMD) is the most prevalent inherited myopathy affecting children, caused by genetic loss of the gene encoding the dystrophin protein. Here we have investigated the use of the <em>Staphylococcus aureus</em> CRISPR-Cas9 system and a...

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מידע ביבליוגרפי
Main Authors:	Hanson, B, Stenler, S, Ahlskog, N, Chwalenia, K, Svrzikapa, N, Coenen-Stass, AML, Weinberg, MS, Wood, MJA, Roberts, TC
פורמט:	Journal article
שפה:	English
יצא לאור:	Cell Press 2022

פריטים דומים

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Immunogold confirmation that utrophin is localized to the normal position of dystrophin in dystrophin-negative transgenic mouse muscle.
מאת: Culle, M, et al.
יצא לאור: (2001)

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

The utrophin and dystrophin genes share similarities in genomic structure.
מאת: Pearce, M, et al.
יצא לאור: (1993)

Utrophin Compensates dystrophin Loss during Mouse Spermatogenesis
מאת: Hung-Chih Chen, et al.
יצא לאור: (2017-08-01)

Functional substitution by TAT-utrophin in dystrophin-deficient mice.
מאת: Kevin J Sonnemann, et al.
יצא לאור: (2009-05-01)

Generation of clusters of dystrophin revertant fibers requires muscle regeneration and tests the function of dystrophin/utrophin transgenes
מאת: Yokota, T, et al.
יצא לאור: (2004)

Confirmation by immunogold labeling that utrophin is localised to the normal position of dystrophin in dystrophin-negative transgenic mouse muscle
מאת: Cullen, M, et al.
יצא לאור: (2001)

The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy
מאת: Guiraud, S, et al.
יצא לאור: (2019)

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

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מאת: Goyenvalle, A, et al.
יצא לאור: (2009)

A quantitative study of bioenergetics in skeletal muscle lacking utrophin and dystrophin.
מאת: Cole, M, et al.
יצא לאור: (2002)

Long-term dystrophin restoration supports development of splice correction therapy for DMD patients with exon 2 duplications
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יצא לאור: (2023-12-01)

Prevention of the dystrophic phenotype in dystrophin/utrophin-deficient muscle following adenovirus-mediated transfer of a utrophin minigene.
מאת: Wakefield, P, et al.
יצא לאור: (2000)

ZZ domain is essentially required for the physiological binding of dystrophin and utrophin to beta-dystroglycan.
מאת: Ishikawa-Sakurai, M, et al.
יצא לאור: (2004)

Dystrophin and utrophin influence fiber type composition and post-synaptic membrane structure.
מאת: Rafael, J, et al.
יצא לאור: (2000)

Metabolic profiles of dystrophin and utrophin expression in mouse models of Duchenne muscular dystrophy.
מאת: Griffin, J, et al.
יצא לאור: (2002)

Promising therapeutic approaches of utrophin replacing dystrophin in the treatment of Duchenne muscular dystrophy
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יצא לאור: (2022-11-01)

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

Mechanism of Deletion Removing All Dystrophin Exons in a Canine Model for DMD Implicates Concerted Evolution of X Chromosome Pseudogenes
מאת: D. Jake VanBelzen, et al.
יצא לאור: (2017-03-01)

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

A Novel CRISPR-Cas9 Strategy to Target DYSTROPHIN Mutations Downstream of Exon 44 in Patient-Specific DMD iPSCs
מאת: Neha R. Dhoke, et al.
יצא לאור: (2024-06-01)

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

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

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

Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin.
מאת: Li, D, et al.
יצא לאור: (2010)

Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin
מאת: Li, D, et al.
יצא לאור: (2010)

Activation of calcineurin and stress activated protein kinase/p38-mitogen activated protein kinase in hearts of utrophin-dystrophin knockout mice.
מאת: Nakamura, A, et al.
יצא לאור: (2001)

Targeting EP2 Receptor Improves Muscle and Bone Health in Dystrophin<sup>−/−</sup>/Utrophin<sup>−/−</sup> Double-Knockout Mice
מאת: Xueqin Gao, et al.
יצא לאור: (2025-01-01)

Neuronal nitric oxide synthase-rescue of dystrophin/utrophin double knockout mice does not require nNOS localization to the cell membrane.
מאת: Michelle Wehling-Henricks, et al.
יצא לאור: (2011-01-01)

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

Exon exchange approach to repair Duchenne dystrophin transcripts.
מאת: Stéphanie Lorain, et al.
יצא לאור: (2010-01-01)

Dystrophin immunogenicity and requirement in myogenic cells: Paradigm shift in gene therapy for DMD
מאת: Dariusz C. Górecki
יצא לאור: (2022-11-01)

Microarray analysis of mdx mice expressing high levels of utrophin: therapeutic implications for dystrophin deficiency.
מאת: Baban, D, et al.
יצא לאור: (2008)

Glycine administration attenuates progression of dystrophic pathology in prednisolone-treated dystrophin/utrophin null mice
מאת: Daniel J. Ham, et al.
יצא לאור: (2019-09-01)

Metabolic remodeling of dystrophic skeletal muscle reveals biological roles for dystrophin and utrophin in adaptation and plasticity
מאת: Justin P. Hardee, et al.
יצא לאור: (2021-03-01)

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

Phenotypic features of dystrophin gene knockout pigs harboring a human artificial chromosome containing the entire dystrophin gene
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