Generation of human myogenic progenitors from pluripotent stem cells for in vivo regeneration

Kim, Hyunkee; Perlingeiro, Rita C.R.

doi:10.1007/s00018-022-04434-8

Cited by 6 publications

(3 citation statements)

References 95 publications

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“…Since this study was carried out with total extracts from crude muscle preparations, low-abundance markers were not detected. However, increases in the myogenic marker molecules CD34 [73][74][75] and cadherin-13 [71,72,137] were identified in the dystrophic mdx-4cv diaphragm by mass spectrometric analysis. The surface marker CD34 was recently shown to exhibit considerable potential as a satellite cell-linked biomarker of skeletal muscle aging [75].…”

Section: Discussionmentioning

confidence: 99%

Proteomic Identification of Markers of Membrane Repair, Regeneration and Fibrosis in the Aged and Dystrophic Diaphragm

Gargan

Dowling

Zweyer

et al. 2022

Life

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Deficiency in the membrane cytoskeletal protein dystrophin is the underlying cause of the progressive muscle wasting disease named Duchenne muscular dystrophy. In order to detect novel disease marker candidates and confirm the complexity of the pathobiochemical signature of dystrophinopathy, mass spectrometric screening approaches represent ideal tools for comprehensive biomarker discovery studies. In this report, we describe the comparative proteomic analysis of young versus aged diaphragm muscles from wild type versus the dystrophic mdx-4cv mouse model of X-linked muscular dystrophy. The survey confirmed the drastic reduction of the dystrophin-glycoprotein complex in the mdx-4cv diaphragm muscle and concomitant age-dependent changes in key markers of muscular dystrophy, including proteins involved in cytoskeletal organization, metabolite transportation, the cellular stress response and excitation-contraction coupling. Importantly, proteomic markers of the regulation of membrane repair, tissue regeneration and reactive myofibrosis were detected by mass spectrometry and changes in key proteins were confirmed by immunoblotting. Potential disease marker candidates include various isoforms of annexin, the matricellular protein periostin and a large number of collagens. Alterations in these proteoforms can be useful to evaluate adaptive, compensatory and pathobiochemical changes in the intracellular cytoskeleton, myofiber membrane integrity and the extracellular matrix in dystrophin-deficient skeletal muscle tissues.

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Section: Discussionmentioning

confidence: 99%

Proteomic Identification of Markers of Membrane Repair, Regeneration and Fibrosis in the Aged and Dystrophic Diaphragm

Gargan

Dowling

Zweyer

et al. 2022

Life

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“…Cell transplantation has long been seen as a potential therapy for DMD [ 88 ] and was recently reviewed [ 89 , 90 , 91 , 92 ]. Issues specific to this therapy are identifying the best muscle regenerating cell type, immune rejection of the donated cells, immune rejection of dystrophin and insufficient engraftment.…”

Section: Therapeutic Strategiesmentioning

confidence: 99%

A Brief Review of Duchenne Muscular Dystrophy Treatment Options, with an Emphasis on Two Novel Strategies

Heydemann

Siemionow

2023

Biomedicines

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Despite the full cloning of the Dystrophin cDNA 35 years ago, no effective treatment exists for the Duchenne Muscular Dystrophy (DMD) patients who have a mutation in this gene. Many treatment options have been considered, investigated preclinically and some clinically, but none have circumvented all barriers and effectively treated the disease without burdening the patients with severe side-effects. However, currently, many novel therapies are in the pipelines of research labs and pharmaceutical companies and many of these have progressed to clinical trials. A brief review of these promising therapies is presented, followed by a description of two novel technologies that when utilized together effectively treat the disease in the mdx mouse model. One novel technology is to generate chimeric cells from the patient’s own cells and a normal donor. The other technology is to systemically transplant these cells into the femur via the intraosseous route.

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“…HMB has been administered to patients afflicted with muscle atrophy arising from debilitating illnesses like AIDS, cancer, and chronic obstructive pulmonary disease (COPD) [ 180 ]. Increasing muscle protein synthesis leads to the suppression of adverse effects of osteoporosis and corticosteroids [ 181 ]. The inhibitory effects of HMB on the NF-κB pathway are achieved by diminishing IL17 expression.…”

Section: Therapeutic Agents Used For Dmd Treatmentmentioning

confidence: 99%

Does β-Hydroxy-β-Methylbutyrate Have Any Potential to Support the Treatment of Duchenne Muscular Dystrophy in Humans and Animals?

Gorji,

Ostaszewski,

Urbańska

et al. 2023

Biomedicines

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Skeletal muscle is the protein reservoir of our body and an important regulator of glucose and lipid homeostasis. The dystrophin gene is the largest gene and has a key role in skeletal muscle construction and function. Mutations in the dystrophin gene cause Duchenne and Becker muscular dystrophy in humans, mice, dogs, and cats. Duchenne muscular dystrophy (DMD) is an X-linked neuromuscular condition causing progressive muscle weakness and premature death. β-hydroxy β-methylbutyrate (HMB) prevents deleterious muscle responses under pathological conditions, including tumor and chronic steroid therapy-related muscle losses. The use of HMB as a dietary supplement allows for increasing lean weight gain; has a positive immunostimulatory effect; is associated with decreased mortality; and attenuates sarcopenia in elderly animals and individuals. This study aimed to identify some genes, metabolic pathways, and biological processes which are common for DMD and HMB based on existing literature and then discuss the consequences of that interaction.

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Generation of human myogenic progenitors from pluripotent stem cells for in vivo regeneration

Cited by 6 publications

References 95 publications

Proteomic Identification of Markers of Membrane Repair, Regeneration and Fibrosis in the Aged and Dystrophic Diaphragm

Proteomic Identification of Markers of Membrane Repair, Regeneration and Fibrosis in the Aged and Dystrophic Diaphragm

A Brief Review of Duchenne Muscular Dystrophy Treatment Options, with an Emphasis on Two Novel Strategies

Does β-Hydroxy-β-Methylbutyrate Have Any Potential to Support the Treatment of Duchenne Muscular Dystrophy in Humans and Animals?

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