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Literature DB >> 26136844

Duchenne muscular dystrophy: current cell therapies.

Dorota Sienkiewicz¹, Wojciech Kulak², Bożena Okurowska-Zawada¹, Grażyna Paszko-Patej¹, Katarzyna Kawnik¹.

Abstract

Duchenne muscular dystrophy is a genetically determined X-linked disease and the most common, progressive pediatric muscle disorder. For decades, research has been conducted to find an effective therapy. This review presents current therapeutic methods for Duchenne muscular dystrophy, based on scientific articles in English published mainly in the period 2000 to 2014. We used the PubMed database to identify and review the most important studies. An analysis of contemporary studies of stem cell therapy and the use of granulocyte colony-stimulating factor (G-CSF) in muscular dystrophy was performed.

Entities: Disease Gene

Keywords: Duchenne muscular dystrophy; G-CSF; stem cells

Year: 2015 PMID： 26136844 PMCID： PMC4480531 DOI： 10.1177/1756285615586123

Source DB: PubMed Journal: Ther Adv Neurol Disord ISSN： 1756-2856 Impact factor: 6.570

85 in total

1. The development of antisense oligonucleotide therapies for Duchenne muscular dystrophy: report on a TREAT-NMD workshop hosted by the European Medicines Agency (EMA), on September 25th 2009.

Authors: F Muntoni
Journal: Neuromuscul Disord Date: 2010-03-26 Impact factor: 4.296

2. Progression of spinal deformity in wheelchair-dependent patients with Duchenne muscular dystrophy who are not treated with steroids: coronal plane (scoliosis) and sagittal plane (kyphosis, lordosis) deformity.

Authors: F Shapiro; D Zurakowski; T Bui; B T Darras
Journal: Bone Joint J Date: 2014-01 Impact factor: 5.082

3. Isolation of multipotent adult stem cells from the dermis of mammalian skin.

Authors: J G Toma; M Akhavan; K J Fernandes; F Barnabé-Heider; A Sadikot; D R Kaplan; F D Miller
Journal: Nat Cell Biol Date: 2001-09 Impact factor: 28.824

Review 4. The muscular dystrophies.

Authors: Alan E H Emery
Journal: Lancet Date: 2002-02-23 Impact factor: 79.321

5. Myoblast implantation in Duchenne muscular dystrophy: the San Francisco study.

Authors: R G Miller; K R Sharma; G K Pavlath; E Gussoni; M Mynhier; A M Lanctot; C M Greco; L Steinman; H M Blau
Journal: Muscle Nerve Date: 1997-04 Impact factor: 3.217

6. Neuroprotective effects of mesenchymal stem cells derived from human embryonic stem cells in transient focal cerebral ischemia in rats.

Authors: Yi-Ping Liu; Hakan Seçkin; Yusuf Izci; Zhong Wei Du; Yi-Ping Yan; Mustafa K Başkaya
Journal: J Cereb Blood Flow Metab Date: 2009-02-11 Impact factor: 6.200

Review 7. Cell based therapy for Duchenne muscular dystrophy.

Authors: Andrea Farini; Paola Razini; Silvia Erratico; Yvan Torrente; Mirella Meregalli
Journal: J Cell Physiol Date: 2009-12 Impact factor: 6.384

Review 8. Neurodegenerative diseases and oxidative stress.

Authors: Kevin J Barnham; Colin L Masters; Ashley I Bush
Journal: Nat Rev Drug Discov Date: 2004-03 Impact factor: 84.694

Review 9. Genetic treatments in muscular dystrophies.

Authors: Francesco Muntoni; Dominic Wells
Journal: Curr Opin Neurol Date: 2007-10 Impact factor: 5.710

10. Early neurodevelopmental assessment in Duchenne muscular dystrophy.

Authors: Marika Pane; Roberta Scalise; Angela Berardinelli; Grazia D'Angelo; Valeria Ricotti; Paolo Alfieri; Isabella Moroni; Louise Hartley; Maria Carmela Pera; Giovanni Baranello; Michela Catteruccia; Tiziana Casalino; Domenico M Romeo; Alessandra Graziano; Claudia Gandioli; Flaviana Bianco; Elena Stacy Mazzone; Maria Elena Lombardo; Mariacristina Scoto; Serena Sivo; Concetta Palermo; Francesca Gualandi; Maria Pia Sormani; Alessandra Ferlini; Enrico Bertini; Francesco Muntoni; Eugenio Mercuri
Journal: Neuromuscul Disord Date: 2013-03-25 Impact factor: 4.296

21 in total

1. Anti-apoptotic Effects of Human Wharton's Jelly-derived Mesenchymal Stem Cells on Skeletal Muscle Cells Mediated via Secretion of XCL1.

Authors: SooJin Kwon; Soo Mi Ki; Sang Eon Park; Min-Jeong Kim; Brian Hyung; Na Kyung Lee; Sangmi Shim; Byung-Ok Choi; Duk L Na; Ji Eun Lee; Jong Wook Chang
Journal: Mol Ther Date: 2016-06-23 Impact factor: 11.454

Review 2. Mesenchymal Stem Cells in the Musculoskeletal System: From Animal Models to Human Tissue Regeneration?

Authors: Klemen Čamernik; Ariana Barlič; Matej Drobnič; Janja Marc; Matjaž Jeras; Janja Zupan
Journal: Stem Cell Rev Rep Date: 2018-06 Impact factor: 5.739

3. In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-epigenetic Modulation.

Authors: Hsin-Kai Liao; Fumiyuki Hatanaka; Toshikazu Araoka; Pradeep Reddy; Min-Zu Wu; Yinghui Sui; Takayoshi Yamauchi; Masahiro Sakurai; David D O'Keefe; Estrella Núñez-Delicado; Pedro Guillen; Josep M Campistol; Cheng-Jang Wu; Li-Fan Lu; Concepcion Rodriguez Esteban; Juan Carlos Izpisua Belmonte
Journal: Cell Date: 2017-12-07 Impact factor: 41.582

4. Purification and Transplantation of Myogenic Progenitor Cell Derived Exosomes to Improve Cardiac Function in Duchenne Muscular Dystrophic Mice.

Authors: Xuan Su; Yan Shen; Yue Jin; Meng Jiang; Neal Weintraub; Yaoliang Tang
Journal: J Vis Exp Date: 2019-04-10 Impact factor: 1.355

5. Long-Term Protective Effect of Human Dystrophin Expressing Chimeric (DEC) Cell Therapy on Amelioration of Function of Cardiac, Respiratory and Skeletal Muscles in Duchenne Muscular Dystrophy.

Authors: Maria Siemionow; Paulina Langa; Sonia Brodowska; Katarzyna Kozlowska; Kristina Zalants; Katarzyna Budzynska; Ahlke Heydemann
Journal: Stem Cell Rev Rep Date: 2022-05-19 Impact factor: 6.692

6. Creation of Dystrophin Expressing Chimeric Cells of Myoblast Origin as a Novel Stem Cell Based Therapy for Duchenne Muscular Dystrophy.

Authors: M Siemionow; J Cwykiel; A Heydemann; J Garcia-Martinez; K Siemionow; E Szilagyi
Journal: Stem Cell Rev Rep Date: 2018-04 Impact factor: 5.739