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...

書誌詳細
主要な著者:	Hanson, B, Stenler, S, Ahlskog, N, Chwalenia, K, Svrzikapa, N, Coenen-Stass, AML, Weinberg, MS, Wood, MJA, Roberts, TC
フォーマット:	Journal article
言語:	English
出版事項:	Cell Press 2022

類似資料

Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD
著者:: Britt Hanson, 等
出版事項: (2022-12-01)

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)

Marginal level dystrophin expression improves clinical outcome in a strain of dystrophin/utrophin double knockout mice.
著者:: Dejia Li, 等
出版事項: (2010-12-01)

Immunogold confirmation that utrophin is localized to the normal position of dystrophin in dystrophin-negative transgenic mouse muscle.
著者:: Culle, M, 等
出版事項: (2001)

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

The utrophin and dystrophin genes share similarities in genomic structure.
著者:: Pearce, M, 等
出版事項: (1993)

Utrophin Compensates dystrophin Loss during Mouse Spermatogenesis
著者:: Hung-Chih Chen, 等
出版事項: (2017-08-01)

Functional substitution by TAT-utrophin in dystrophin-deficient mice.
著者:: Kevin J Sonnemann, 等
出版事項: (2009-05-01)

Generation of clusters of dystrophin revertant fibers requires muscle regeneration and tests the function of dystrophin/utrophin transgenes
著者:: Yokota, T, 等
出版事項: (2004)

Confirmation by immunogold labeling that utrophin is localised to the normal position of dystrophin in dystrophin-negative transgenic mouse muscle
著者:: Cullen, M, 等
出版事項: (2001)

The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy
著者:: Guiraud, S, 等
出版事項: (2019)

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)

Upgrading U7snRNA To Complete Efficient Rescue of Dystrophin by Exon-Skipping in DMD Patients
著者:: Goyenvalle, A, 等
出版事項: (2009)

A quantitative study of bioenergetics in skeletal muscle lacking utrophin and dystrophin.
著者:: Cole, M, 等
出版事項: (2002)

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

Prevention of the dystrophic phenotype in dystrophin/utrophin-deficient muscle following adenovirus-mediated transfer of a utrophin minigene.
著者:: Wakefield, P, 等
出版事項: (2000)

ZZ domain is essentially required for the physiological binding of dystrophin and utrophin to beta-dystroglycan.
著者:: Ishikawa-Sakurai, M, 等
出版事項: (2004)

Dystrophin and utrophin influence fiber type composition and post-synaptic membrane structure.
著者:: Rafael, J, 等
出版事項: (2000)

Metabolic profiles of dystrophin and utrophin expression in mouse models of Duchenne muscular dystrophy.
著者:: Griffin, J, 等
出版事項: (2002)

Promising therapeutic approaches of utrophin replacing dystrophin in the treatment of Duchenne muscular dystrophy
著者:: Ruo Wu, 等
出版事項: (2022-11-01)

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

Mechanism of Deletion Removing All Dystrophin Exons in a Canine Model for DMD Implicates Concerted Evolution of X Chromosome Pseudogenes
著者:: D. Jake VanBelzen, 等
出版事項: (2017-03-01)

Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts.
著者:: Joshua Lee, 等
出版事項: (2018-01-01)

A Novel CRISPR-Cas9 Strategy to Target DYSTROPHIN Mutations Downstream of Exon 44 in Patient-Specific DMD iPSCs
著者:: Neha R. Dhoke, 等
出版事項: (2024-06-01)

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)

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)

Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin.
著者:: Li, D, 等
出版事項: (2010)

Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin
著者:: Li, D, 等
出版事項: (2010)

Activation of calcineurin and stress activated protein kinase/p38-mitogen activated protein kinase in hearts of utrophin-dystrophin knockout mice.
著者:: Nakamura, A, 等
出版事項: (2001)

Targeting EP2 Receptor Improves Muscle and Bone Health in Dystrophin<sup>−/−</sup>/Utrophin<sup>−/−</sup> Double-Knockout Mice
著者:: Xueqin Gao, 等
出版事項: (2025-01-01)

Neuronal nitric oxide synthase-rescue of dystrophin/utrophin double knockout mice does not require nNOS localization to the cell membrane.
著者:: Michelle Wehling-Henricks, 等
出版事項: (2011-01-01)

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)

Exon exchange approach to repair Duchenne dystrophin transcripts.
著者:: Stéphanie Lorain, 等
出版事項: (2010-01-01)

Dystrophin immunogenicity and requirement in myogenic cells: Paradigm shift in gene therapy for DMD
著者:: Dariusz C. Górecki
出版事項: (2022-11-01)

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)

Glycine administration attenuates progression of dystrophic pathology in prednisolone-treated dystrophin/utrophin null mice
著者:: Daniel J. Ham, 等
出版事項: (2019-09-01)

Metabolic remodeling of dystrophic skeletal muscle reveals biological roles for dystrophin and utrophin in adaptation and plasticity
著者:: Justin P. Hardee, 等
出版事項: (2021-03-01)

A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.
著者:: Yin, H, 等
出版事項: (2009)

Phenotypic features of dystrophin gene knockout pigs harboring a human artificial chromosome containing the entire dystrophin gene
著者:: Masahito Watanabe, 等
出版事項: (2023-09-01)