Results 101 to 110 of about 7,812 (251)

Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model

Molecular Therapy: Methods & Clinical Development, 2023
Micro-dystrophin gene replacement therapies for Duchenne muscular dystrophy (DMD) are currently in clinical trials, but have not been thoroughly investigated for their efficacy on cardiomyopathy progression to heart failure. We previously validated Fiona/
Arden B. Piepho, Jeovanna Lowe, Laurel R. Cumby, Lisa E. Dorn, Dana M. Lake, Neha Rastogi, Megan D. Gertzen, Sarah L. Sturgill, Guy L. Odom, Mark T. Ziolo, Federica Accornero, Jeffrey S. Chamberlain, Jill A. Rafael-Fortney   +12 more
doaj  

Efficacy of Prednisolone in Generated Myotubes Derived From Fibroblasts of Duchenne Muscular Dystrophy Patients

Frontiers in Pharmacology, 2018
Duchenne muscular dystrophy (DMD) is a recessive X-linked form of muscular dystrophy characterized by progressive muscle degeneration. This disease is caused by the mutation or deletion of the dystrophin gene. Currently, there are no effective treatments
Tsubasa Kameyama, Tsubasa Kameyama, Kazuki Ohuchi, Kazuki Ohuchi, Michinori Funato, Shiori Ando, Shiori Ando, Satoshi Inagaki, Satoshi Inagaki, Arisu Sato, Arisu Sato, Junko Seki, Chizuru Kawase, Kazuhiro Tsuruma, Ichizo Nishino, Shinsuke Nakamura, Masamitsu Shimazawa, Takashi Saito, Shin’ichi Takeda, Hideo Kaneko, Hideaki Hara   +20 more
doaj   +1 more source

Muscle‐Derived Bioactive Factors: MyoEVs and Myokines

Cell Proliferation, Volume 58, Issue 3, March 2025.
Overview of the functions and applications of myokines and MyoEVs.
Xupeng Liu, Ziyue Yao, Liping Zhang, Ng Shyh‐Chang   +3 more
wiley   +1 more source

The spectrin family of proteins: a unique coiled-coil fold for various molecular surface properties

, 2014
International audienceThe spectrin superfamily is composed of proteins involved in cytolinker functions. Their main structural feature is a large central subdomain with numerous repeats folded in triple helical coiled-coils.
Delalande, Olivier, Hubert, Jean-François, Le Rumeur, Elisabeth, Nicolas, Aurélie   +3 more
core   +2 more sources

High Throughput Screening in Duchenne Muscular Dystrophy: From Drug Discovery to Functional Genomics

Biology, 2014
Centers for the screening of biologically active compounds and genomic libraries are becoming common in the academic setting and have enabled researchers devoted to developing strategies for the treatment of diseases or interested in studying a ...
Thomas J.J. Gintjee, Alvin S.H. Magh, Carmen Bertoni   +2 more
doaj   +1 more source

Extracellular matrix: Dystroglycan interactions—Roles for the dystrophin‐associated glycoprotein complex in skeletal tissue dynamics

International Journal of Experimental Pathology, Volume 106, Issue 2, March 2025.
Abstract Contributions made by the dystrophin‐associated glycoprotein complex (DGC) to cell–cell and cell‐extracellular matrix (ECM) interactions are vital in development, homeostasis and pathobiology. This review explores how DGC functions may extend to skeletal pathophysiology by appraising the known roles of its major ECM ligands, and likely ...
Mark Hopkinson, Andrew A. Pitsillides
wiley   +1 more source

How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse [PDF]

, 2015
Splice modulation therapy has shown great clinical promise in Duchenne muscular dystrophy, resulting in the production of dystrophin protein. Despite this, the relationship between restoring dystrophin to established dystrophic muscle and its ability to ...
Betts, C, Coursindel, T, El Andaloussi, S, Gait, M J, Godfrey, C, Hammond, S, Hildyard, J C W, McClorey, G, Muses, S, O'Donovan, L, Terry, R L, Wells, D J, Wells, K E, Wood, M J   +13 more
core   +1 more source

Gene therapy for genetic diseases: challenges and future directions

MedComm, Volume 6, Issue 2, February 2025.
The graphical abstract provides an overview of gene therapy approaches, detailing the components of the therapy and the various delivery routes. Both in vivo and ex vivo strategies facilitate the implementation of gene replacement, gene suppression, gene supplementation, and gene editing.
Beibei Qie, Jianghua Tuo, Feilong Chen, Haili Ding, Lei Lyu   +4 more
wiley   +1 more source

N-terminal α Dystroglycan (αDG-N): A Potential Serum Biomarker for Duchenne Muscular Dystrophy [PDF]

, 2018
Biological Sciences: 2nd Place (The Ohio State University Edward F. Hayes Graduate Research Forum)Background: Duchenne Muscular Dystrophy (DMD) is a severe, progressive, neuromuscular disorder of childhood.
Crowe, Kelly E.
core   +1 more source

Macrophages producing chondroitin sulfate proteoglycan‐4 induce neuro‐cardiac junction impairment in Duchenne muscular dystrophy

The Journal of Pathology, Volume 265, Issue 1, Page 1-13, January 2025.
Abstract Duchenne muscular dystrophy (DMD) is caused by the absence of the full form of the dystrophin protein, which is essential for maintaining the structural integrity of muscle cells, including those in the heart and respiratory system. Despite progress in understanding the molecular mechanisms associated with DMD, myocardial insufficiency ...
Marika Milan, Fabio Maiullari, Maila Chirivì, Maria Grazia Ceraolo, Rebecca Zigiotto, Andrea Soluri, Silvia Maiullari, Elisa Landoni, Dario Di Silvestre, Francesca Brambilla, Pierluigi Mauri, Veronica De Paolis, Nicole Fratini, Maria Cristina Crosti, Chiara Cordiglieri, Chiara Parisi, Antonella Calogero, Dror Seliktar, Yvan Torrente, Chiara Lanzuolo, Gianpietro Dotti, Mirco Toccafondi, Mauro Bombaci, Elena De Falco, Claudia Bearzi, Roberto Rizzi   +25 more
wiley   +1 more source