Systemic MyoAAV-saRNA delivery activates endogenous utrophin and rescues dystrophic pathology in mdx mice [PDF]
Regenerative TherapyBackground: Therapeutic activation of endogenous utrophin, a homolog of dystrophin, represents a mutation-agnostic therapeutic strategy with considerable potential for treating Duchenne muscular dystrophy (DMD).
Zhikai Xiahou +8 more
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Activation of endogenous full-length utrophin by MyoAAV-UA as a therapeutic approach for Duchenne muscular dystrophy [PDF]
Nature CommunicationsActivation of endogenous full-length utrophin, a dystrophin homolog, presents an attractive therapeutic strategy for Duchenne muscular dystrophy (DMD), regardless of mutation types and loci.
Ruo Wu +18 more
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Dystrophin Restorative and Compensatory Gene Addition Therapies for Duchenne Muscular Dystrophy: Could CRISPRa Provide a Realistic Alternative? [PDF]
MusclesDuchenne muscular dystrophy (DMD), which results from mutations that disrupt the expression of dystrophin proteins, is characterized by progressive muscle fiber wasting and the development of skeletal muscle fibrosis.
Zakaria Rostamitehrani +2 more
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L-Arginine Activates the Neuregulin-1/ErbB Receptor Signaling Pathway and Increases Utrophin mRNA Levels in C2C12 Cells [PDF]
Biochemistry Research InternationalL-arginine induces the expression of utrophin in skeletal muscle cells, so it has been proposed as a pharmacological treatment to attenuate the symptoms of Duchenne muscular dystrophy (DMD).
Gladys Tapia +6 more
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Skeletal muscle‐specific PGC‐1α‐b overexpression prevents eccentric contraction‐induced muscle injury through an utrophin‐independent pathway in mice [PDF]
Physiological ReportsSlower oxidative fibers are more resistant to eccentric contraction (ECC)‐induced muscle damage than fast‐twitch glycolytic fibers, but the mechanisms remain unclear. This study investigated the roles of the exercise‐inducible PGC‐1α isoform PGC‐1α‐b and
Azuma Naito +7 more
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Biallelic variants in the UTRN gene cause a novel form of multiple congenital arthrogryposis [PDF]
Frontiers in GeneticsArthrogryposis multiplex congenita (AMC) is a large group of congenital conditions characterized by joint contractures affecting two or more body areas.
Evgeniya Melnik +9 more
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Respiratory pathology in the mdx/utrn -/- mouse: A murine model for Duchenne Muscular Dystrophy (DMD). [PDF]
PLoS ONEDuchenne muscular dystrophy (DMD) is an X-linked devastating disease caused by a lack of dystrophin which results in progressive muscle weakness. As muscle weakness progresses, respiratory insufficiency and hypoventilation result in significant morbidity
Marán Y Hernández Rodríguez +10 more
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Fundamental Research, 2022 Duchenne muscular dystrophy (DMD) is a serious genetic neuromuscular rare disease that is prevalent and caused by the mutation/deletion of the X-linked DMD gene that encodes dystrophin.
Ruo Wu +3 more
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Cis-Acting Sequence Elements and Upstream Open Reading Frame in Mouse Utrophin-A 5'-UTR Repress Cap-Dependent Translation. [PDF]
PLoS ONE, 2015 Utrophin, the autosomal homologue of dystrophin can functionally compensate for dystrophin deficiency. Utrophin upregulation could therefore be a therapeutic strategy in Duchenne Muscular Dystrophy (DMD) that arises from mutation in dystrophin gene.
Trinath Ghosh, Utpal Basu
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Differential requirement for utrophin in the induced pluripotent stem cell correction of muscle versus fat in muscular dystrophy mice. [PDF]
PLoS ONE, 2011 Duchenne muscular dystrophy (DMD) is an incurable degenerative muscle disorder. We injected WT mouse induced pluripotent stem cells (iPSCs) into mdx and mdx∶utrophin mutant blastocysts, which are predisposed to develop DMD with an increasing degree of ...
Amanda J Beck +9 more
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