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...

Descripció completa

Dades bibliogràfiques
Autors principals:	Hanson, B, Stenler, S, Ahlskog, N, Chwalenia, K, Svrzikapa, N, Coenen-Stass, AML, Weinberg, MS, Wood, MJA, Roberts, TC
Format:	Journal article
Idioma:	English
Publicat:	Cell Press 2022

Ítems similars

Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD
per: Britt Hanson, et al.
Publicat: (2022-12-01)

Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties
per: Chwalenia, K, et al.
Publicat: (2022)

Marginal level dystrophin expression improves clinical outcome in a strain of dystrophin/utrophin double knockout mice.
per: Dejia Li, et al.
Publicat: (2010-12-01)

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

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

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

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

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

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

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

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

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

Upgrading U7snRNA To Complete Efficient Rescue of Dystrophin by Exon-Skipping in DMD Patients
per: Goyenvalle, A, et al.
Publicat: (2009)

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

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

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

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

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

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

Promising therapeutic approaches of utrophin replacing dystrophin in the treatment of Duchenne muscular dystrophy
per: Ruo Wu, et al.
Publicat: (2022-11-01)

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Phenotypic features of dystrophin gene knockout pigs harboring a human artificial chromosome containing the entire dystrophin gene
per: Masahito Watanabe, et al.
Publicat: (2023-09-01)