Autologous skeletal muscle derived cells expressing a novel functional dystrophin provide a potential therapy for Duchenne Muscular Dystrophy
Published 2016 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
Autologous skeletal muscle derived cells expressing a novel functional dystrophin provide a potential therapy for Duchenne Muscular Dystrophy
Authors
Keywords
-
Journal
Scientific Reports
Volume 6, Issue 1, Pages -
Publisher
Springer Nature
Online
2016-01-27
DOI
10.1038/srep19750
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD)
- (2015) Klaudia Kawecka et al. CURRENT GENE THERAPY
- How much dystrophin is enough: the physiological consequences of different levels of dystrophin in themdxmouse
- (2015) Caroline Godfrey et al. HUMAN MOLECULAR GENETICS
- Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus
- (2015) Yongping Yue et al. HUMAN MOLECULAR GENETICS
- Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division
- (2015) Nicolas A Dumont et al. NATURE MEDICINE
- The Human Desmin Promoter Drives Robust Gene Expression for Skeletal Muscle Stem Cell-Mediated Gene Therapy
- (2014) Jacqueline Jonuschies et al. CURRENT GENE THERAPY
- Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease
- (2014) Olivier Negre et al. CURRENT GENE THERAPY
- Uncovering and Dissecting the Genotoxicity of Self-inactivating Lentiviral Vectors In Vivo
- (2014) Daniela Cesana et al. MOLECULAR THERAPY
- Human Skeletal Muscle–derived CD133+ Cells Form Functional Satellite Cells After Intramuscular Transplantation in Immunodeficient Host Mice
- (2014) Jinhong Meng et al. MOLECULAR THERAPY
- Green fluorescent protein expression triggers proteome changes in breast cancer cells
- (2013) J.V.F. Coumans et al. EXPERIMENTAL CELL RESEARCH
- Identification of New Dystroglycan Complexes in Skeletal Muscle
- (2013) Eric K. Johnson et al. PLoS One
- Revertant Fibers in the mdx Murine Model of Duchenne Muscular Dystrophy: An Age- and Muscle-Related Reappraisal
- (2013) Sarah R. Pigozzo et al. PLoS One
- A New Immuno-, Dystrophin-Deficient Model, theNSG-mdx4CvMouse, Provides Evidence for Functional Improvement Following Allogeneic Satellite Cell Transplantation
- (2013) Robert W. Arpke et al. STEM CELLS
- Evidence-based path to newborn screening for duchenne muscular dystrophy
- (2012) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- Understanding lentiviral vector chromatin targeting: working to reduce insertional mutagenic potential for gene therapy
- (2012) C Papayannakos et al. GENE THERAPY
- CD47Low Status on CD4 Effectors Is Necessary for the Contraction/Resolution of the Immune Response in Humans and Mice
- (2012) Vu Quang Van et al. PLoS One
- 2 and 3 helices of dystrophin R16 and R17 frame a microdomain in the 1 helix of dystrophin R17 for neuronal NOS binding
- (2012) Y. Lai et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection
- (2011) A. Biffi et al. BLOOD
- Novel Mini–Dystrophin Gene Dual Adeno-Associated Virus Vectors Restore Neuronal Nitric Oxide Synthase Expression at the Sarcolemma
- (2011) Yadong Zhang et al. HUMAN GENE THERAPY
- Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
- (2011) Sebahattin Cirak et al. LANCET
- Restoration of the Dystrophin-associated Glycoprotein Complex After Exon Skipping Therapy in Duchenne Muscular Dystrophy
- (2011) Sebahattin Cirak et al. MOLECULAR THERAPY
- Contribution of Human Muscle-Derived Cells to Skeletal Muscle Regeneration in Dystrophic Host Mice
- (2011) Jinhong Meng et al. PLoS One
- Physiological Characterization of Muscle Strength With Variable Levels of Dystrophin Restoration in mdx Mice Following Local Antisense Therapy
- (2010) Paul S Sharp et al. MOLECULAR THERAPY
- The contribution of human synovial stem cells to skeletal muscle regeneration
- (2010) Jinhong Meng et al. NEUROMUSCULAR DISORDERS
- Marginal Level Dystrophin Expression Improves Clinical Outcome in a Strain of Dystrophin/Utrophin Double Knockout Mice
- (2010) Dejia Li et al. PLoS One
- Sub-physiological sarcoglycan expression contributes to compensatory muscle protection in mdx mice
- (2009) Dejia Li et al. HUMAN MOLECULAR GENETICS
- Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy
- (2009) Yi Lai et al. JOURNAL OF CLINICAL INVESTIGATION
- In Vivo Myogenic Potential of Human CD133+ Muscle-derived Stem Cells: A Quantitative Study
- (2009) Elisa Negroni et al. MOLECULAR THERAPY
- Desmin-regulated Lentiviral Vectors for Skeletal Muscle Gene Transfer
- (2009) Gillian E Talbot et al. MOLECULAR THERAPY
- Dystrophin Delivery to Muscles of mdx Mice Using Lentiviral Vectors Leads to Myogenic Progenitor Targeting and Stable Gene Expression
- (2009) En Kimura et al. MOLECULAR THERAPY
- Preservation of Muscle Force in Mdx3cv Mice Correlates with Low-Level Expression of a Near Full-Length Dystrophin Protein
- (2008) Dejia Li et al. AMERICAN JOURNAL OF PATHOLOGY
- Highly Efficient, Functional Engraftment of Skeletal Muscle Stem Cells in Dystrophic Muscles
- (2008) Massimiliano Cerletti et al. CELL
Publish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn MoreFind the ideal target journal for your manuscript
Explore over 38,000 international journals covering a vast array of academic fields.
Search