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...

وصف كامل

التفاصيل البيبلوغرافية
المؤلفون الرئيسيون:	Hanson, B, Stenler, S, Ahlskog, N, Chwalenia, K, Svrzikapa, N, Coenen-Stass, AML, Weinberg, MS, Wood, MJA, Roberts, TC
التنسيق:	Journal article
اللغة:	English
منشور في:	Cell Press 2022

مواد مشابهة

Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD
حسب: Britt Hanson, وآخرون
منشور في: (2022-12-01)

Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties
حسب: Chwalenia, K, وآخرون
منشور في: (2022)

Marginal level dystrophin expression improves clinical outcome in a strain of dystrophin/utrophin double knockout mice.
حسب: Dejia Li, وآخرون
منشور في: (2010-12-01)

Immunogold confirmation that utrophin is localized to the normal position of dystrophin in dystrophin-negative transgenic mouse muscle.
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منشور في: (2001)

Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping
حسب: Goyenvalle, A, وآخرون
منشور في: (2010)

The utrophin and dystrophin genes share similarities in genomic structure.
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منشور في: (1993)

Utrophin Compensates dystrophin Loss during Mouse Spermatogenesis
حسب: Hung-Chih Chen, وآخرون
منشور في: (2017-08-01)

Functional substitution by TAT-utrophin in dystrophin-deficient mice.
حسب: Kevin J Sonnemann, وآخرون
منشور في: (2009-05-01)

Generation of clusters of dystrophin revertant fibers requires muscle regeneration and tests the function of dystrophin/utrophin transgenes
حسب: Yokota, T, وآخرون
منشور في: (2004)

Confirmation by immunogold labeling that utrophin is localised to the normal position of dystrophin in dystrophin-negative transgenic mouse muscle
حسب: Cullen, M, وآخرون
منشور في: (2001)

The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy
حسب: Guiraud, S, وآخرون
منشور في: (2019)

Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping.
حسب: Goyenvalle, A, وآخرون
منشور في: (2010)

Upgrading U7snRNA To Complete Efficient Rescue of Dystrophin by Exon-Skipping in DMD Patients
حسب: Goyenvalle, A, وآخرون
منشور في: (2009)

A quantitative study of bioenergetics in skeletal muscle lacking utrophin and dystrophin.
حسب: Cole, M, وآخرون
منشور في: (2002)

Long-term dystrophin restoration supports development of splice correction therapy for DMD patients with exon 2 duplications
حسب: Thomas C. Roberts
منشور في: (2023-12-01)

Prevention of the dystrophic phenotype in dystrophin/utrophin-deficient muscle following adenovirus-mediated transfer of a utrophin minigene.
حسب: Wakefield, P, وآخرون
منشور في: (2000)

ZZ domain is essentially required for the physiological binding of dystrophin and utrophin to beta-dystroglycan.
حسب: Ishikawa-Sakurai, M, وآخرون
منشور في: (2004)

Dystrophin and utrophin influence fiber type composition and post-synaptic membrane structure.
حسب: Rafael, J, وآخرون
منشور في: (2000)

Metabolic profiles of dystrophin and utrophin expression in mouse models of Duchenne muscular dystrophy.
حسب: Griffin, J, وآخرون
منشور في: (2002)

Promising therapeutic approaches of utrophin replacing dystrophin in the treatment of Duchenne muscular dystrophy
حسب: Ruo Wu, وآخرون
منشور في: (2022-11-01)

Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene
حسب: Kane L Greer, وآخرون
منشور في: (2014-01-01)

Mechanism of Deletion Removing All Dystrophin Exons in a Canine Model for DMD Implicates Concerted Evolution of X Chromosome Pseudogenes
حسب: D. Jake VanBelzen, وآخرون
منشور في: (2017-03-01)

Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts.
حسب: Joshua Lee, وآخرون
منشور في: (2018-01-01)

A Novel CRISPR-Cas9 Strategy to Target DYSTROPHIN Mutations Downstream of Exon 44 in Patient-Specific DMD iPSCs
حسب: Neha R. Dhoke, وآخرون
منشور في: (2024-06-01)

Effective exon skipping and dystrophin restoration by 2'-o-methoxyethyl antisense oligonucleotide in dystrophin-deficient mice.
حسب: Lu Yang, وآخرون
منشور في: (2013-01-01)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping
حسب: Goyenvalle, A, وآخرون
منشور في: (2012)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping.
حسب: Goyenvalle, A, وآخرون
منشور في: (2012)

Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin.
حسب: Li, D, وآخرون
منشور في: (2010)

Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin
حسب: Li, D, وآخرون
منشور في: (2010)

Activation of calcineurin and stress activated protein kinase/p38-mitogen activated protein kinase in hearts of utrophin-dystrophin knockout mice.
حسب: Nakamura, A, وآخرون
منشور في: (2001)

Targeting EP2 Receptor Improves Muscle and Bone Health in Dystrophin<sup>−/−</sup>/Utrophin<sup>−/−</sup> Double-Knockout Mice
حسب: Xueqin Gao, وآخرون
منشور في: (2025-01-01)

Neuronal nitric oxide synthase-rescue of dystrophin/utrophin double knockout mice does not require nNOS localization to the cell membrane.
حسب: Michelle Wehling-Henricks, وآخرون
منشور في: (2011-01-01)

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

Exon exchange approach to repair Duchenne dystrophin transcripts.
حسب: Stéphanie Lorain, وآخرون
منشور في: (2010-01-01)

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

Microarray analysis of mdx mice expressing high levels of utrophin: therapeutic implications for dystrophin deficiency.
حسب: Baban, D, وآخرون
منشور في: (2008)

Glycine administration attenuates progression of dystrophic pathology in prednisolone-treated dystrophin/utrophin null mice
حسب: Daniel J. Ham, وآخرون
منشور في: (2019-09-01)

Metabolic remodeling of dystrophic skeletal muscle reveals biological roles for dystrophin and utrophin in adaptation and plasticity
حسب: Justin P. Hardee, وآخرون
منشور في: (2021-03-01)

A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.
حسب: Yin, H, وآخرون
منشور في: (2009)

Phenotypic features of dystrophin gene knockout pigs harboring a human artificial chromosome containing the entire dystrophin gene
حسب: Masahito Watanabe, وآخرون
منشور في: (2023-09-01)