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Condition: Muscular Dystrophy

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Total 22 results found since Jan 2013.

Fine Tuning of Phosphorothioate Inclusion in 2 ′-O-Methyl Oligonucleotides Contributes to Specific Cell Targeting for Splice-Switching Modulation
Splice-switching antisense oligonucleotide- (SSO-) mediated correction of framedisrupting mutation-containing premessenger RNA (mRNA) transcripts using exon skipping is a highly promising treatment method for muscular diseases such as Duchenne muscular dystrophy (DMD). Phosphorothioate (PS) chemistry, a commonly used oligonucleotide modification, has been shown to increase the stability of and improve the pharmacokinetics of SSOs. However, the effect of PS inclusion in 2′-O-methyl SSOs (2OMe) on cellular uptake and splice switching is less well-understood. At present, we demonstrate that the modification of PS facilitate...
Source: Frontiers in Physiology - October 13, 2021 Category: Physiology Source Type: research

Suppression of MyoD induces spontaneous adipogenesis in skeletal muscle progenitor cell culture
AbstractThe degree of intramuscular adipose tissue accumulation is one of the factors affecting meat quality. Accumulation of adipocytes is also observed under the pathological condition of skeletal muscle such as muscular dystrophy and sarcopenia. The origin of adipocytes seen in skeletal muscle is mesenchymal progenitor cells that can give rise to both adipocytes and fibroblasts. In the present study, we demonstrated that siRNA-mediated suppression of MyoD expression in rat skeletal muscle progenitor cell culture, which comprises both myogenic satellite cells and mesenchymal progenitor cells, resulted in diminished myotu...
Source: Animal Science Journal - July 7, 2021 Category: Zoology Authors: Keitaro Yamanouchi, Katsuyuki Nakamura, Shiho Takeuchi, Tohru Hosoyama, Takashi Matsuwaki, Masugi Nishihara Tags: RESEARCH ARTICLE Source Type: research

Limb Girdle Muscular Dystrophy and Therapy: Current progress in Cell and Gene-based approaches.
Abstract The limb-girdle muscular dystrophies (LGMD) are genetically heterogeneous disorders, responsible for muscle wasting and severe form of dystrophies. Despite the critical developments in the insight and information of pathomechanisms of limb-girdle muscular dystrophy, any definitive treatments do not exist, and current strategies are only based on the improvement of the signs of disorder and to enhance the life quality without resolving an underlying cause. There is a crucial relationship between pharmacological therapy and different consequences; therefore, other treatment strategies will be required. New ...
Source: Current Gene Therapy - February 16, 2020 Category: Genetics & Stem Cells Authors: Taheri F, Taghizadeh E, Pour MJR, Rostami D, Renani PG, Rastgar-Moghadam A, Hayat SMG Tags: Curr Gene Ther Source Type: research

The JAK/STAT Pathway in Skeletal Muscle Pathophysiology
Conclusion and Perspectives The IL-6/JAK/STAT signaling cascade plays a dominant role in skeletal muscle pathophysiology. IL-6 autocrine, paracrine, and endocrine functions assign to its downstream effectors pivotal importance in skeletal muscle-wasting-associated diseases and other multiple system diseases where muscle acts in communication with other organs. Targeting the components of the JAK/STAT pathway recently emerged as a strategic approach for the treatment of inflammatory diseases and human cancer. This review highlights the opposite outcomes on muscle biology caused by the amount of local and systemic release ...
Source: Frontiers in Physiology - April 29, 2019 Category: Physiology Source Type: research

Gene Therapy Leaves a Vicious Cycle
Reena Goswami1, Gayatri Subramanian2, Liliya Silayeva1, Isabelle Newkirk1, Deborah Doctor1, Karan Chawla2, Saurabh Chattopadhyay2, Dhyan Chandra3, Nageswararao Chilukuri1 and Venkaiah Betapudi1,4* 1Neuroscience Branch, Research Division, United States Army Medical Research Institute of Chemical Defense, Aberdeen, MD, United States 2Department of Medical Microbiology and Immunology, University of Toledo College of Medicine and Life Sciences, Toledo, OH, United States 3Roswell Park Comprehensive Cancer Center, Buffalo, NY, United States 4Department of Physiology and Biophysics, Case Western Reserve University, Clev...
Source: Frontiers in Oncology - April 23, 2019 Category: Cancer & Oncology Source Type: research

Oligonucleotides —A Novel Promising Therapeutic Option for IBD
Conclusions In this review, we focused on recent and past approaches to test the therapeutic efficacy of oligonucleotide based therapies in IBD. The combining mechanistic mode of oligonucleotide based therapeutics is a targeted action on specific pro-inflammatory molecules, which are over activated in IBD patients and contribute significantly to disease pathogenesis. The proposed high selectivity of the agents is derived from its mode of action, that aims to specifically block certain inflammatory molecular patterns, without a general systemic effect on other molecular targets. It would be important for each oligonucleot...
Source: Frontiers in Pharmacology - April 23, 2019 Category: Drugs & Pharmacology Source Type: research

Role of Mitogen activated-kinase (MAPK)-phosphatase (MKP)-5 in pulmonary fibrosis
Conclusion: Intact MKP5 is required for induction of changes in lung fibroblasts in-vitro and during bleomycin-induced lung fibrosis in-vivo. MKP5 inhibition represent a promising therapeutic target for experimental and lung fibrosis.
Source: European Respiratory Journal - November 19, 2018 Category: Respiratory Medicine Authors: Tzouvelekis, A., Karampitsakos, T., Min, K., Xylourgidis, N., Yu, G., Herazo-Maya, J., Bizenhofer, L., Bennett, A., Kaminski, N. Tags: Idiopathic interstitial pneumonias Source Type: research

MKP-5 inhibition blunts fibrotic responses in-vitro and in-vivo through negative regulation of TGFB1-induced smad3-signalling
Conclusion: We conclude that intact MKP5 is required for induction of changes in lung fibroblasts in-vitro and during bleomycin-induced lung fibrosis in-vivo. Our results couple MKP5 activity with TGFB1 signaling machinery identifying MKP5 inhibition as a promising therapeutic strategy for experimental and human lung fibrosis.
Source: European Respiratory Journal - November 19, 2018 Category: Respiratory Medicine Authors: Tzouvelekis, A., Xylourgidis, N., Min, K., Karampitsakos, T., Ninou, I., Barbayianni, I., Bennett, A., Aidinis, V., Kaminski, N. Tags: Mechanisms of Lung Injury and Repair Source Type: research

Co-Administration of Myostatin-Targeting siRNA and ActRIIB-Fc Fusion Protein Increases Masseter Muscle Mass and Fiber Size.
Authors: Bayarsaikhan O, Kawai N, Mori H, Kinouchi N, Nikawa T, Tanaka E Abstract Myostatin, a member of the TGF-β superfamily, is a negative regulator of skeletal muscle cell growth and differentiation, and binds with high affinity to the activin type IIB receptor (ActRIIB). The soluble ligand-binding domain of ActRIIB fused to the Fc domain of IgG (ActRIIB-Fc) potently binds and inhibits TGF-β family members in muscle, leading to rapid and marked muscle growth. The present study was designed to assess the effectiveness of the co-delivery of myostatin-targeting siRNA (Mstn-siRNA) and ActRIIB-Fc into skeletal mus...
Source: Journal of Nutritional Science and Vitaminology - October 7, 2017 Category: Nutrition Tags: J Nutr Sci Vitaminol (Tokyo) Source Type: research

Ret function in muscle stem cells points to tyrosine kinase inhibitor therapy for facioscapulohumeral muscular dystrophy
Facioscapulohumeral muscular dystrophy (FSHD) involves sporadic expression of DUX4, which inhibits myogenesis and is pro-apoptotic. To identify target genes, we over-expressed DUX4 in myoblasts and found that the receptor tyrosine kinaseRet was significantly up-regulated, suggesting a role in FSHD.RET is dynamically expressed during myogenic progression in mouse and human myoblasts. Constitutive expression of eitherRET9 orRET51 increased myoblast proliferation, whereas siRNA-mediated knockdown ofRet induced myogenic differentiation. Suppressing RET activity using Sunitinib, a clinically-approved tyrosine kinase inhibitor, ...
Source: eLife - November 14, 2016 Category: Biomedical Science Tags: Cell Biology DUX4 facioscapulohumeral muscular dystrophy FSHD Human Human Biology and Medicine Mouse RET Sunitinib therapy Source Type: research

Maladaptative autophagy impairs adipose function in Congenital Generalized Lipodystrophy due to cavin-1 deficiency.
CONCLUSIONS: This study showed that cavin-1 deficiency resulted in maladaptative autophagy which contributed to insulin resistance and altered adipocyte differentiation. These new pathophysiological mechanisms could open new therapeutic perspectives for adipose tissue diseases including CGL4. PMID: 27144934 [PubMed - as supplied by publisher]
Source: The Journal of Clinical Endocrinology and Metabolism - May 3, 2016 Category: Endocrinology Authors: Salle-Teyssières L, Auclair M, Terro F, Nemani M, Elsayed SM, Elsobky E, Lathrop M, Délépine M, Lascols O, Capeau J, Magré J, Vigouroux C Tags: J Clin Endocrinol Metab Source Type: research

{mu}-Crystallin controls muscle function through thyroid hormone action Research Communication
In this study, Crym was preferentially expressed in skeletal muscle throughout the body. Crym-knockout mice exhibited a significant hypertrophy of fast-twitch glycolytic type IIb fibers, causing an increase in grip strength and high intensity running ability in Crym-null mice. Genetic inactivation of Crym or blockade of Crym by siRNA-mediated knockdown up-regulated the gene expression of fast-glycolytic contractile fibers in satellite cell-derived myotubes in vitro. These alterations in Crym-inactivated muscle were rescued by inhibition of thyroid hormone, even though Crym is a positive regulator of thyroid hormone action ...
Source: FASEB Journal - April 28, 2016 Category: Biology Authors: Seko, D., Ogawa, S., Li, T.-S., Taimura, A., Ono, Y. Tags: Research Communication Source Type: research

TGF-β1 activates the canonical NF-κB signaling to promote cell survival and proliferation in dystrophic muscle fibroblasts In Vitro.
Abstract Activated fibroblasts continue to proliferate at injury sites, leading to progressive muscular fibrosis in Duchenne muscular dystrophy (DMD). TGF-β1 is a dominant profibrotic mediator thought to play a critical role in muscle fibrosis; however, the implicated mechanisms are not fully understood. Here we showed that TGF-β1 increased the resistance to apoptosis and stimulated cell cycle progression in dystrophic muscle fibroblasts under serum deprivation conditions in vitro. TGF-β1 treatment activated the canonical NF-κB pathway; and we found that pharmacological inhibition of IKKβ with IMD-0354 and Re...
Source: Biochemical and Biophysical Research communications - February 10, 2016 Category: Biochemistry Authors: Ma ZY, Zhong ZG, Qiu MY, Zhong YH, Zhang WX Tags: Biochem Biophys Res Commun Source Type: research

Loss of epigenetic silencing of the DUX4 transcription factor gene in facioscapulohumeral muscular dystrophy
Current genetic and molecular evidence best supports an epigenetic mechanism for facioscapulohumeral muscular dystrophy (FSHD), whereby de-repression of the D4Z4 macrosatellite array leads to aberrant expression of the DUX4 transcription factor in skeletal muscle. This de-repression is triggered by either array contraction or (more rarely) by mutation of the SMCHD1 (structural maintenance of chromosomes flexible hinge domain containing 1) gene. Activation of DUX4 targets, including germline genes and several mammalian retrotransposons, then drives pathogenesis. A direct role for DUX4 mRNA in suppression of nonsense-mediate...
Source: Human Molecular Genetics - September 16, 2015 Category: Genetics & Stem Cells Authors: Hewitt, J. E. Tags: INVITED REVIEWS Source Type: research