Results 11 to 20 of about 56,165 (274)

Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models. [PDF]

Nat Med, 2019
Song Y, Morales L, Malik AS, Mead AF, Greer CD, Mitchell MA, Petrov MT, Su LT, Choi ME, Rosenblum ST, Lu X, VanBelzen DJ, Krishnankutty RK, Balzer FJ, Loro E, French R, Propert KJ, Zhou S, Kozyak BW, Nghiem PP, Khurana TS, Kornegay JN, Stedman HH.   +22 more
europepmc   +2 more sources

Dlk1-Dio3 cluster miRNAs regulate mitochondrial functions in the dystrophic muscle in Duchenne muscular dystrophy

Life Science Alliance, 2023
A large number of DLK1-DIO3 miRNAs are up-regulated in the muscles and the serum of Duchenne muscular dystrophy, animal models, and patients. Mitochondrial functions, and in particular oxidative phosphorylation, are targeted by these coordinately up ...
Ai Vu Hong, Nathalie Bourg, Peggy Sanatine, Jerome Poupiot, Karine Charton, Evelyne Gicquel, Emmanuelle Massourides, Marco Spinazzi, Isabelle Richard, David Israeli   +9 more
doaj   +1 more source

The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development [PDF]

, 2015
Despite multiple publications on potential therapies for neuromuscular diseases (NMD) in cell and animal models only a handful reach clinical trials. The ability to prioritise drug development according to objective criteria is particularly critical in ...
Allen, Hugh, Bourke, John, Burghes, Arthur, Bushby, Kate, Buyse, Gunnar, Caizergues, Didier, Catlin, Nick, Clemens, Paula, Cnaan, Avital, Comi, Giacomo, Connor, Edward, Csimma, Cristina, Day, Simon, de Luca, Annamaria, de Montleau, Béatrice, de Visser, Marianne, Dittrich, Sven, Dubrosky, Alberto, Eagle, Michelle, Finkel, Richard, Fishbeck, Kenneth, Flanigan, Kevin M., Furlong, Patricia, Grounds, Miranda, Hauschke, Dieter, Heslop, Emma, Hesterlee, Sharon, Hoffman, Eric, Irwin, Joseph, Jarecki, Jill, Kaufmann, Petra, Kelly, Michael, Korinthenberg, Rudolf, Laforêt, Pascal, Larkindale, Jane, Lovering, Richard, Mayer, Henry, McCall, John, McDonald, Robert, McNally, Elizabeth, McNeil, Dawn Elizabeth, McNeil, Elizabeth, Mendell, Jerry, Miller, Debra, Nagaraju, Kanneboyina, North, Kathryn, Ouillade, Marie-Christine, Straub, Volker, Wagner, Kathryn R., Wells, Dominic J.   +49 more
core   +8 more sources

Duchenne Muscular Dystrophy Animal Models

, 2022
Duchenne muscular dystrophy is a complex and severe orphan disease. It develops when the organism lacks the expression of dystrophin - a large structural protein. Dystrophin is transcribed from the largest gene in the human genome. At the moment, there is no cure available. Dozens of groups all over the world search for cure.
V., Egorova, Tatiana, I., Galkin, an, V., Ivanova, Yulia, V., Polikarpova, Anna   +3 more
openaire   +3 more sources

Loss of MyoD Promotes Fate Transdifferentiation of Myoblasts Into Brown Adipocytes

EBioMedicine, 2017
Brown adipose tissue (BAT) represents a promising agent to ameliorate obesity and other metabolic disorders. However, the abundance of BAT decreases with age and BAT paucity is a common feature of obese subjects. As brown adipocytes and myoblasts share a
Chao Wang, Weiyi Liu, Yaohui Nie, Mulan Qaher, Hannah Elizabeth Horton, Feng Yue, Atsushi Asakura, Shihuan Kuang   +7 more
doaj   +1 more source

Profiles of Steroid Hormones in Canine X-Linked Muscular Dystrophy via Stable Isotope Dilution LC-MS/MS. [PDF]

PLoS ONE, 2015
Golden retriever muscular dystrophy (GRMD) provides the best animal model for characterizing the disease progress of the human disorder, Duchenne muscular dystrophy (DMD).
Helio A Martins-Júnior, Rosineide C Simas, Marina P Brolio, Christina R Ferreira, Felipe Perecin, Guilherme de P Nogueira, Maria A Miglino, Daniele S Martins, Marcos N Eberlin, Carlos E Ambrósio   +9 more
doaj   +1 more source

Myocardial Contractile Dysfunction is Present Without Histopathology in a Mouse Model of Limb-Girdle Muscular Dystrophy-2F and is Prevented after Claudin-5 Virotherapy

Frontiers in Physiology, 2016
Mutations in several members of the dystrophin glycoprotein complex lead to skeletal and cardiomyopathies. Cardiac care for these muscular dystrophies consists of management of symptoms with standard heart medications after detection of reduced whole ...
Nima Milani-Nejad, Eric J Schultz, Jessica L Slabaugh, Paul M L Janssen, Jill A. Rafael-Fortney   +4 more
doaj   +1 more source

Circadian Genes as Exploratory Biomarkers in DMD: Results From Both the mdx Mouse Model and Patients

Frontiers in Physiology, 2021
Duchenne muscular dystrophy (DMD) is a rare genetic disease due to dystrophin gene mutations which cause progressive weakness and muscle wasting. Circadian rhythm coordinates biological processes with the 24-h cycle and it plays a key role in maintaining
Rachele Rossi, Rachele Rossi, Maria Sofia Falzarano, Hana Osman, Hana Osman, Annarita Armaroli, Chiara Scotton, Paola Mantuano, Brigida Boccanegra, Ornella Cappellari, Elena Schwartz, Anton Yuryev, Eugenio Mercuri, Enrico Bertini, Adele D’Amico, Marina Mora, Camilla Johansson, Cristina Al-Khalili Szigyarto, Cristina Al-Khalili Szigyarto, Annamaria De Luca, Alessandra Ferlini, Alessandra Ferlini   +21 more
doaj   +1 more source

Identification of muscle-specific microRNAs in serum of muscular dystrophy animal models: promising novel blood-based markers for muscular dystrophy. [PDF]

PLoS ONE, 2011
Duchenne muscular dystrophy (DMD) is a lethal X-linked disorder caused by mutations in the dystrophin gene, which encodes a cytoskeletal protein, dystrophin. Creatine kinase (CK) is generally used as a blood-based biomarker for muscular disease including
Hideya Mizuno, Akinori Nakamura, Yoshitsugu Aoki, Naoki Ito, Soichiro Kishi, Kazuhiro Yamamoto, Masayuki Sekiguchi, Shin'ichi Takeda, Kazuo Hashido   +8 more
doaj   +1 more source

Investigating synthetic oligonucleotide targeting of miR31 in Duchenne muscular dystrophy [PDF]

, 2016
Exon-skipping via synthetic antisense oligonucleotides represents one of the most promising potential therapies for Duchenne muscular dystrophy (DMD), yet this approach is highly sequence-specific and thus each oligonucleotide is of benefit to only a ...
Hildyard, J C W, Wells, D J
core   +1 more source