Results 11 to 20 of about 25,343 (235)

Spatiotemporal analysis of dystrophin expression during muscle repair [PDF]

Skeletal Muscle
Background Dystrophin mRNA is produced from a very large genetic locus and transcription of a single mRNA requires approximately 16 h. This prolonged interval between initiation and completion results in unusual transcriptional behaviour: in skeletal ...
John C.W. Hildyard, Liberty E. Roskrow, Dominic J. Wells, Richard J. Piercy   +3 more
doaj   +2 more sources

Dystrophin and dystrophin-associated protein in muscles and nerves from monkey

European Journal of Histochemistry, 2009
Since all organs (i.e. skeletal, cardiac, smooth muscles and sciatic nerve) are never only taken from a single patient, all these tissues were obtained from one cynomolgus monkey, a model closely resembling humans.
M Royuela, D Chazalette, F Rivier, G Hugon, R Paniagua, V Guerlavais, JA Fehrentz, J Martinez, D Mornet   +8 more
doaj   +7 more sources

A combinatorial oligonucleotide therapy to improve dystrophin restoration and dystrophin-deficient muscle health [PDF]

Molecular Therapy: Nucleic Acids
Despite the proven safety of dystrophin-targeting phosphorodiamidate morpholino oligomer (PMO) therapy, poor delivery of the PMOs limit the efficacy of this dystrophin restoring gene therapy for Duchenne muscular dystrophy (DMD).
Young Jae Moon, Ravi Hindupur, Iteoluwakishi H. Gamu, Nikki M. McCormack, Fatima Shaikh, James S. Novak, Jyoti K. Jaiswal   +6 more
doaj   +2 more sources

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

Science Translational Medicine, 2017
Leonela Amoasii, Chengzu Long, Hui Li
exaly   +2 more sources

In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice [PDF]

Circulation Research, 2017
Mona El Refaey, Benjamin D Canan, David E Symer   +2 more
exaly   +2 more sources

Current Trends in Duchenne Muscular Dystrophy Research and Therapy: 3D Cardiac Modelling. [PDF]

J Cachexia Sarcopenia Muscle
ABSTRACT Duchenne muscular dystrophy (DMD), caused by dystrophin deficiency, presents a multifaceted challenge that affects both skeletal muscle function and cardiomyocyte homeostasis, causing progressive degeneration and life‐threatening cardiac complications by adolescence.
Przymuszała M, Białobrzeska M, Dulak J, Florczyk-Soluch U.   +3 more
europepmc   +2 more sources

Full-length human dystrophin on human artificial chromosome compensates for mouse dystrophin deficiency in a Duchenne muscular dystrophy mouse model

Scientific Reports, 2023
Dystrophin maintains membrane integrity as a sarcolemmal protein. Dystrophin mutations lead to Duchenne muscular dystrophy, an X-linked recessive disorder.
Yosuke Hiramuki, Satoshi Abe, Narumi Uno, Kanako Kazuki, Shuta Takata, Hitomaru Miyamoto, Haruka Takayama, Kayoko Morimoto, Shoko Takehara, Mitsuhiko Osaki, Jun Tanihata, Shin’ichi Takeda, Kazuma Tomizuka, Mitsuo Oshimura, Yasuhiro Kazuki   +14 more
doaj   +1 more source

The effects of low levels of dystrophin on mouse muscle function and pathology. [PDF]

PLoS ONE, 2012
Duchenne muscular dystrophy (DMD) is a severe progressive muscular disorder caused by reading frame disrupting mutations in the DMD gene, preventing the synthesis of functional dystrophin. As dystrophin provides muscle fiber stability during contractions,
Maaike van Putten, Margriet Hulsker, Vishna Devi Nadarajah, Sandra H van Heiningen, Ella van Huizen, Maarten van Iterson, Peter Admiraal, Tobias Messemaker, Johan T den Dunnen, Peter A C 't Hoen, Annemieke Aartsma-Rus   +10 more
doaj   +1 more source

A high–throughput digital script for multiplexed immunofluorescent analysis and quantification of sarcolemmal and sarcomeric proteins in muscular dystrophies

Acta Neuropathologica Communications, 2020
The primary molecular endpoint for many Duchenne muscular dystrophy (DMD) clinical trials is the induction, or increase in production, of dystrophin protein in striated muscle.
Dominic Scaglioni, Matthew Ellis, Francesco Catapano, Silvia Torelli, Darren Chambers, Lucy Feng, Caroline Sewry, Jennifer Morgan, Francesco Muntoni, Rahul Phadke   +9 more
doaj   +1 more source

Proteomic analysis reveals new cardiac-specific dystrophin-associated proteins. [PDF]

PLoS ONE, 2012
Mutations affecting the expression of dystrophin result in progressive loss of skeletal muscle function and cardiomyopathy leading to early mortality.
Eric K Johnson, Liwen Zhang, Marvin E Adams, Alistair Phillips, Michael A Freitas, Stanley C Froehner, Kari B Green-Church, Federica Montanaro   +7 more
doaj   +1 more source