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myoblast transplantation DMD

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https://www.readbyqxmd.com/read/26941360/induction-of-ccaat-enhancer-binding-protein-%C3%AE-expression-with-the-phosphodiesterase-inhibitor-isobutylmethylxanthine-improves-myoblast-engraftment-into-dystrophic-muscle
#1
Neena Lala-Tabbert, Dechen Fu, Nadine Wiper-Bergeron
UNLABELLED: Duchenne muscular dystrophy (DMD), caused by mutations in the dystrophin gene, is the most common muscular dystrophy. Characterized by rounds of muscle degeneration and regeneration, DMD features progressive muscle wasting and is fatal. One approach for treatment is transplantation of muscle progenitor cells to repair and restore dystrophin expression to damaged muscle. However, the success of this approach has been limited by difficulties in isolating large numbers of myogenic progenitors with strong regenerative potential, poor engraftment, poor survival of donor cells, and limited migration in the diseased muscle...
April 2016: Stem Cells Translational Medicine
https://www.readbyqxmd.com/read/26223276/mmp1-gene-expression-enhances-myoblast-migration-and-engraftment-following-implanting-into-mdx-scid-mice
#2
Haiying Pan, Kinga Vojnits, Thomas T Liu, Fanwei Meng, Lei Yang, Yigang Wang, Johnny Huard, Charles S Cox, Kevin P Lally, Yong Li
Myoblast transplantation (MT) is a method to introduce healthy genes into abnormal skeletal muscle. It has been considered as a therapeutic modality in the last few decades for diseases such as Duchenne Muscular Dystrophy (DMD). However, challenges including cell death and poor graft engraftment have limited its application. The current experiment utilizes MMP1 gene transfer to improve the efficacy of myoblast transplantation into the diseased dystrophic skeletal muscle of mdx mice. Our results indicated that MMP1 expression can promote myogenic differentiation and fusion capacities, increase migration of MMP1 expressing myoblasts in vitro, as well as improve engraftment of dystrophin positive myofibers in vivo...
2015: Cell Adhesion & Migration
https://www.readbyqxmd.com/read/25955813/cell-therapy-in-duchenne-muscular-dystrophy-treatment-clinical-trials-overview
#3
REVIEW
Anna Bajek, Dorota Porowinska, Tomasz Kloskowski, Edyta Brzoska, Maria A Ciemerych, Tomasz Drewa
Duchenne muscular dystrophy (DMD), the most common and most severe form of all muscular dystrophies, leads to progressive muscle fiber necrosis, fibroblast proliferation, and growth of fibrous tissue and fat. The most common cause of death in DMD patients is cardiac and respiratory failure. Current pharmacological and other treatment methods do not lead to full recovery. For this reason, new alternatives for skeletal muscle regeneration are being investigated. Transplantation of myoblasts from healthy donors is one studied approach to muscle treatment in DMD patients...
2015: Critical Reviews in Eukaryotic Gene Expression
https://www.readbyqxmd.com/read/25692716/multiplex-crispr-cas9-based-genome-editing-for-correction-of-dystrophin-mutations-that-cause-duchenne-muscular-dystrophy
#4
David G Ousterout, Ami M Kabadi, Pratiksha I Thakore, William H Majoros, Timothy E Reddy, Charles A Gersbach
The CRISPR/Cas9 genome-editing platform is a promising technology to correct the genetic basis of hereditary diseases. The versatility, efficiency and multiplexing capabilities of the CRISPR/Cas9 system enable a variety of otherwise challenging gene correction strategies. Here, we use the CRISPR/Cas9 system to restore the expression of the dystrophin gene in cells carrying dystrophin mutations that cause Duchenne muscular dystrophy (DMD). We design single or multiplexed sgRNAs to restore the dystrophin reading frame by targeting the mutational hotspot at exons 45-55 and introducing shifts within exons or deleting one or more exons...
February 18, 2015: Nature Communications
https://www.readbyqxmd.com/read/25492562/correction-of-dystrophin-expression-in-cells-from-duchenne-muscular-dystrophy-patients-through-genomic-excision-of-exon-51-by-zinc-finger-nucleases
#5
David G Ousterout, Ami M Kabadi, Pratiksha I Thakore, Pablo Perez-Pinera, Matthew T Brown, William H Majoros, Timothy E Reddy, Charles A Gersbach
Duchenne muscular dystrophy (DMD) is caused by genetic mutations that result in the absence of dystrophin protein expression. Oligonucleotide-induced exon skipping can restore the dystrophin reading frame and protein production. However, this requires continuous drug administration and may not generate complete skipping of the targeted exon. In this study, we apply genome editing with zinc finger nucleases (ZFNs) to permanently remove essential splicing sequences in exon 51 of the dystrophin gene and thereby exclude exon 51 from the resulting dystrophin transcript...
March 2015: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/24331373/myod-mediates-skeletal-myogenic-differentiation-of-human-amniotic-fluid-stem-cells-and-regeneration-of-muscle-injury
#6
Ju Ang Kim, Yun Hee Shon, Jeong Ok Lim, James J Yoo, Hong-In Shin, Eui Kyun Park
INTRODUCTION: Human amniotic fluid stem (hAFS) cells have been shown to differentiate into multiple lineages, including myoblasts. However, molecular mechanisms underlying the myogenic differentiation of hAFS cells and their regenerative potential for muscle injury remain to be elucidated. METHODS: In order to induce myogenic differentiation of hAFS cells, lentiviruses for MYOD were constructed and transduced into hAFS cells. Formation of myotube-like cells was analyzed by immunocytochemistry, and expression of molecular markers for myoblasts was analyzed by reverse transcription polymerase chain reaction and Western blotting...
2013: Stem Cell Research & Therapy
https://www.readbyqxmd.com/read/23977226/matrix-metalloproteinase-9-inhibition-improves-proliferation-and-engraftment-of-myogenic-cells-in-dystrophic-muscle-of-mdx-mice
#7
Sajedah M Hindi, Jonghyun Shin, Yuji Ogura, Hong Li, Ashok Kumar
Duchenne muscular dystrophy (DMD) caused by loss of cytoskeletal protein dystrophin is a devastating disorder of skeletal muscle. Primary deficiency of dystrophin leads to several secondary pathological changes including fiber degeneration and regeneration, extracellular matrix breakdown, inflammation, and fibrosis. Matrix metalloproteinases (MMPs) are a group of extracellular proteases that are involved in tissue remodeling, inflammation, and development of interstitial fibrosis in many disease states. We have recently reported that the inhibition of MMP-9 improves myopathy and augments myofiber regeneration in mdx mice (a mouse model of DMD)...
2013: PloS One
https://www.readbyqxmd.com/read/23731976/assessment-of-a-symptomatic-duchenne-muscular-dystrophy-carrier-20-years-after-myoblast-transplantation-from-her-asymptomatic-identical-twin-sister
#8
Jean-Yves Hogrel, Fabien Zagnoli, Aurélie Canal, Bodvael Fraysse, Jean-Pierre Bouchard, Daniel Skuk, Michel Fardeau, Jacques P Tremblay
Because it is due to a mutation on the X-chromosome, Duchenne muscular dystrophy rarely affects women, unless there is an unequal lyonisation of the X-chromosome containing the normal dystrophin gene. We report here the unique situation of a symptomatic Duchenne muscular dystrophy woman who was transplanted with myoblasts received from her asymptomatic monozygotic twin sister 20 years ago. Specific dynamometry was performed to possibly detect a long-term effect of this cell therapy. Long-term safety of myoblast transplantation was established by this exceptional case...
July 2013: Neuromuscular Disorders: NMD
https://www.readbyqxmd.com/read/23568004/molecular-imaging-to-target-transplanted-muscle-progenitor-cells
#9
Kelly Gutpell, Rebecca McGirr, Lisa Hoffman
Duchenne muscular dystrophy (DMD) is a severe genetic neuromuscular disorder that affects 1 in 3,500 boys, and is characterized by progressive muscle degeneration. In patients, the ability of resident muscle satellite cells (SCs) to regenerate damaged myofibers becomes increasingly inefficient. Therefore, transplantation of muscle progenitor cells (MPCs)/myoblasts from healthy subjects is a promising therapeutic approach to DMD. A major limitation to the use of stem cell therapy, however, is a lack of reliable imaging technologies for long-term monitoring of implanted cells, and for evaluating its effectiveness...
2013: Journal of Visualized Experiments: JoVE
https://www.readbyqxmd.com/read/23336007/muscle-side-population-cells-from-dystrophic-or-injured-muscle-adopt-a-fibro-adipogenic-fate
#10
Christopher M Penton, Jennifer M Thomas-Ahner, Eric K Johnson, Cynthia McAllister, Federica Montanaro
Muscle side population (SP) cells are rare multipotent stem cells that can participate in myogenesis and muscle regeneration upon transplantation. While they have been primarily studied for the development of cell-based therapies for Duchenne muscular dystrophy, little is known regarding their non-muscle lineage choices or whether the dystrophic muscle environment affects their ability to repair muscle. Unfortunately, the study of muscle SP cells has been challenged by their low abundance and the absence of specific SP cell markers...
2013: PloS One
https://www.readbyqxmd.com/read/23031415/low-molecular-weight-dextran-sulfate-binds-to-human-myoblasts-and-improves-their-survival-after-transplantation-in-mice
#11
Thomas Laumonier, Amandine Pradier, Pierre Hoffmeyer, Vincent Kindler, Jacques Menetrey
Myoblast transplantation represents a promising therapeutic strategy in the treatment of several genetic muscular disorders including Duchenne muscular dystrophy. Nevertheless, such an approach is impaired by the rapid death, limited migration, and rejection of transplanted myoblasts by the host. Low molecular weight dextran sulfate (DXS), a sulfated polysaccharide, has been reported to act as a cytoprotectant for various cell types. Therefore, we investigated whether DXS could act as a "myoblastprotectant" either in vitro or in vivo after transplantation in immunodeficient mice...
2013: Cell Transplantation
https://www.readbyqxmd.com/read/22990676/myoblasts-derived-from-normal-hescs-and-dystrophic-hipscs-efficiently-fuse-with-existing-muscle-fibers-following-transplantation
#12
Sébastien Goudenege, Carl Lebel, Nicolas B Huot, Christine Dufour, Isao Fujii, Jean Gekas, Joël Rousseau, Jacques P Tremblay
Human embryonic stem cells (hESCs) and human-induced pluripotent stem cells (hiPSCs) have an endless self-renewal capacity and can theoretically differentiate into all types of lineages. They thus represent an unlimited source of cells for therapies of regenerative diseases, such as Duchenne muscular dystrophy (DMD), and for tissue repair in specific medical fields. However, at the moment, the low number of efficient specific lineage differentiation protocols compromises their use in regenerative medicine. We developed a two-step procedure to differentiate hESCs and dystrophic hiPSCs in myogenic cells...
November 2012: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/22917925/osteopontin-deficiency-delays-inflammatory-infiltration-and-the-onset-of-muscle-regeneration-in-a-mouse-model-of-muscle-injury
#13
Kitipong Uaesoontrachoon, Dimuthu K Wasgewatte Wijesinghe, Eleanor J Mackie, Charles N Pagel
Osteopontin is secreted by skeletal muscle myoblasts and stimulates their proliferation. Expression of osteopontin in skeletal muscle is upregulated in pathological conditions including Duchenne muscular dystrophy, and recent evidence suggests that osteopontin might influence the course of this disease. The current study was undertaken to determine whether osteopontin regulates skeletal muscle regeneration. A whole muscle autografting model of regeneration in osteopontin-null and wild-type mice was used. Osteopontin expression was found to be strongly upregulated in wild-type grafts during the initial degeneration and subsequent early regeneration phases that are observed in this model...
January 2013: Disease Models & Mechanisms
https://www.readbyqxmd.com/read/22745439/transplantation-of-genetically-corrected-human-ipsc-derived-progenitors-in-mice-with-limb-girdle-muscular-dystrophy
#14
Francesco Saverio Tedesco, Mattia F M Gerli, Laura Perani, Sara Benedetti, Federica Ungaro, Marco Cassano, Stefania Antonini, Enrico Tagliafico, Valentina Artusi, Emanuela Longa, Rossana Tonlorenzi, Martina Ragazzi, Giorgia Calderazzi, Hidetoshi Hoshiya, Ornella Cappellari, Marina Mora, Benedikt Schoser, Peter Schneiderat, Mitsuo Oshimura, Roberto Bottinelli, Maurilio Sampaolesi, Yvan Torrente, Vania Broccoli, Giulio Cossu
Mesoangioblasts are stem/progenitor cells derived from a subset of pericytes found in muscle that express alkaline phosphatase. They have been shown to ameliorate the disease phenotypes of different animal models of muscular dystrophy and are now undergoing clinical testing in children affected by Duchenne's muscular dystrophy. Here, we show that patients with a related disease, limb-girdle muscular dystrophy 2D (LGMD2D), which is caused by mutations in the gene encoding α-sarcoglycan, have reduced numbers of this pericyte subset and thus produce too few mesoangioblasts for use in autologous cell therapy...
June 27, 2012: Science Translational Medicine
https://www.readbyqxmd.com/read/22449443/administration-of-a-soluble-activin-type-iib-receptor-promotes-the-transplantation-of-human-myoblasts-in-dystrophic-mice
#15
Raouia Fakhfakh, Se-Jin Lee, Jacques P Tremblay
Duchenne muscular dystrophy (DMD) is a recessive disease caused by a dystrophin gene mutation. Myoblast transplantation permits the introduction of the dystrophin gene into dystrophic muscle fibers. However, this strategy has so far produced limited results. Modulation of transforming growth factor-β (TGF-β) superfamily signaling promotes skeletal muscle differentiation and growth and myogenic regeneration. We investigated the possibility that the combination of TGF-β superfamily signaling inhibition with myoblast transplantation might be an effective therapeutic approach in dystrophin-deficient patients...
2012: Cell Transplantation
https://www.readbyqxmd.com/read/22396316/clone-derived-human-af-amniotic-fluid-stem-cells-are-capable-of-skeletal-myogenic-differentiation-in-vitro-and-in-vivo
#16
Xiaorong Ma, Shengli Zhang, Junmei Zhou, Baisong Chen, Yafeng Shang, Tongbing Gao, Xue Wang, Hua Xie, Fang Chen
Stem cell-based therapy may be the most promising method to cure skeletal muscle degenerative diseases such as Duchenne muscular dystrophy (DMD) and trauma in the future. Human amniotic fluid is enriched with early-stage stem cells from developing fetuses and these cells have cardiomyogenic potential both in vitro and in vivo. In the present study, we investigated the characteristics of human amniotic fluid-derived AF-type stem (HAF-AFS) cells by flow cytometry, immunofluorescence staining, reverse-transcription polymerase chain reaction, and osteogenic and adipogenic differentiation analysis...
August 2012: Journal of Tissue Engineering and Regenerative Medicine
https://www.readbyqxmd.com/read/22223500/-induced-pluripotent-stem-ips-cell-based-cell-therapy-for-muscular-dystrophy-current-progress-and-future-prospects
#17
REVIEW
Takashi Nishiyama, Shin'ichi Takeda
Duchenne muscular dystrophy (DMD) is a devastating muscle disorder caused by mutations in the dystrophin gene. There is currently no effective treatment for DMD. Muscle satellite cells are tissue-specific stem cells found in the skeletal muscle; these cells play a central role in postnatal muscle growth and regeneration, and are, therefore, a potential source for stem cell therapy for DMD. However, transplantation of satellite cell-derived myoblasts has not yet been successful in humans. Patient-specific induced pluripotent stem (iPS) cells are expected to be a source for autologous cell transplantation therapy for DMD, because iPS cells can proliferate vigorously in vitro and can differentiate into multiple cell lineages both in vitro and in vivo...
January 2012: Brain and Nerve, Shinkei Kenkyū No Shinpo
https://www.readbyqxmd.com/read/22195027/murine-and-human-myogenic-cells-identified-by-elevated-aldehyde-dehydrogenase-activity-implications-for-muscle-regeneration-and-repair
#18
Joseph B Vella, Seth D Thompson, Mark J Bucsek, Minjung Song, Johnny Huard
BACKGROUND: Despite the initial promise of myoblast transfer therapy to restore dystrophin in Duchenne muscular dystrophy patients, clinical efficacy has been limited, primarily by poor cell survival post-transplantation. Murine muscle derived stem cells (MDSCs) isolated from slowly adhering cells (SACs) via the preplate technique, induce greater muscle regeneration than murine myoblasts, primarily due to improved post-transplantation survival, which is conferred by their increased stress resistance capacity...
2011: PloS One
https://www.readbyqxmd.com/read/22040608/immortalized-pathological-human-myoblasts-towards-a-universal-tool-for-the-study-of-neuromuscular-disorders
#19
Kamel Mamchaoui, Capucine Trollet, Anne Bigot, Elisa Negroni, Soraya Chaouch, Annie Wolff, Prashanth K Kandalla, Solenne Marie, James Di Santo, Jean Lacau St Guily, Francesco Muntoni, Jihee Kim, Susanne Philippi, Simone Spuler, Nicolas Levy, Sergiu C Blumen, Thomas Voit, Woodring E Wright, Ahmed Aamiri, Gillian Butler-Browne, Vincent Mouly
BACKGROUND: Investigations into both the pathophysiology and therapeutic targets in muscle dystrophies have been hampered by the limited proliferative capacity of human myoblasts. Isolation of reliable and stable immortalized cell lines from patient biopsies is a powerful tool for investigating pathological mechanisms, including those associated with muscle aging, and for developing innovative gene-based, cell-based or pharmacological biotherapies. METHODS: Using transduction with both telomerase-expressing and cyclin-dependent kinase 4-expressing vectors, we were able to generate a battery of immortalized human muscle stem-cell lines from patients with various neuromuscular disorders...
2011: Skeletal Muscle
https://www.readbyqxmd.com/read/21903674/alpha-sarcoglycan-is-required-for-fgf-dependent-myogenic-progenitor-cell-proliferation-in-vitro-and-in-vivo
#20
Marco Cassano, Arianna Dellavalle, Francesco Saverio Tedesco, Mattia Quattrocelli, Stefania Crippa, Flavio Ronzoni, Agnese Salvade, Emanuele Berardi, Yvan Torrente, Giulio Cossu, Maurilio Sampaolesi
Mice deficient in α-sarcoglycan (Sgca-null mice) develop progressive muscular dystrophy and serve as a model for human limb girdle muscular dystrophy type 2D. Sgca-null mice suffer a more severe myopathy than that of mdx mice, the model for Duchenne muscular dystrophy. This is the opposite of what is observed in humans and the reason for this is unknown. In an attempt to understand the cellular basis of this severe muscular dystrophy, we isolated clonal populations of myogenic progenitor cells (MPCs), the resident postnatal muscle progenitors of dystrophic and wild-type mice...
October 2011: Development
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