Results 81 to 90 of about 35,595 (240)

Duchenne muscular dystrophy treatment with lentiviral vector containing mini‐dystrophin gene in vivo

MedComm
Duchenne muscular dystrophy (DMD) is an incurable X‐linked recessive genetic disease caused by mutations in the dystrophin gene. Many researchers aim to restore truncated dystrophin via viral vectors.
Xiaoyu Wang, Yanghui Zhu, Taiqing Liu, Lingyan Zhou, Yunhai Fu, Jinhua Zhao, Yinqi Li, Yeteng Zheng, Xiaodong Yang, Xiangjie Di, Yang Yang, Zhiyao He   +11 more
doaj   +1 more source

Promising therapeutic approaches of utrophin replacing dystrophin in the treatment of Duchenne muscular dystrophy

Fundamental Research, 2022
Duchenne muscular dystrophy (DMD) is a serious genetic neuromuscular rare disease that is prevalent and caused by the mutation/deletion of the X-linked DMD gene that encodes dystrophin.
Ruo Wu, Yafeng Song, Shiwen Wu, Yongchang Chen   +3 more
doaj  

Stem cells from umbilical cord blood do have myogenic potential, with and without differentiation induction in vitro

Journal of Translational Medicine, 2009
The dystrophin gene, located at Xp21, codifies dystrophin, which is part of a protein complex responsible for the membrane stability of muscle cells. Its absence on muscle causes Duchenne Muscular Dystrophy (DMD), a severe disorder, while a defect of ...
Gollop Thomaz R, Zucconi Eder, Vieira Natássia M, Secco Mariane, Jazedje Tatiana, Vainzof Mariz, Zatz Mayana   +6 more
doaj   +1 more source

Sequence similarity of the amino-terminal domain of Drosophila beta spectrin to alpha actinin and dystrophin. [PDF]

, 1989
T J Byers, A Husain-Chishti, Ronald R. Dubreuil, Daniel Branton, Lawrence S.B. Goldstein   +4 more
openalex   +1 more source

Cell fractionation studies indicate that dystrophin is a protein of surface membranes of skeletal muscle [PDF]

, 1989
G. Salviati, Romeo Betto, Stefania Ceoldo, E. Biasia, E. Bonilla, Armand F. Miranda, S. DiMauro   +6 more
openalex   +1 more source

Dystrophin contains multiple independent membrane-binding domains.

Human Molecular Genetics, 2016
Dystrophin is a large sub-sarcolemmal protein. Its absence leads to Duchenne muscular dystrophy (DMD). Binding to the sarcolemma is essential for dystrophin to protect muscle from contraction-induced injury. It has long been thought that membrane binding
Junling Zhao, Kasun Kodippili, Y. Yue, Chady H. Hakim, L. Wasala, Xiufang Pan, Keqing Zhang, N. N. Yang, D. Duan, Yi Lai   +9 more
semanticscholar   +1 more source

Dystrophin analysis in the diagnosis of muscular dystrophy. [PDF]

, 1989
Andrew Norman, H E Hughes, D. Gardner‐Medwin, L. V. B. Nicholson   +3 more
openalex   +1 more source

Dystrophin and the brain [PDF]

Developmental Medicine & Child Neurology, 2010
Ricotti, Valeria, Roberts, Roland G., Muntoni, Francesco   +2 more
openaire   +4 more sources

Autologous skeletal muscle derived cells expressing a novel functional dystrophin provide a potential therapy for Duchenne Muscular Dystrophy

Scientific Reports, 2016
Autologous stem cells that have been genetically modified to express dystrophin are a possible means of treating Duchenne Muscular Dystrophy (DMD). To maximize the therapeutic effect, dystrophin construct needs to contain as many functional motifs as ...
J. Meng, J. Counsell, M. Reza, S. Laval, O. Danos, A. Thrasher, Hanns Lochmüller, F. Muntoni, J. Morgan   +8 more
semanticscholar   +1 more source

How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse.

Human Molecular Genetics, 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 ...
C. Godfrey, S. Muses, G. Mcclorey, K. Wells, T. Coursindel, R. Terry, Corinne A Betts, S. Hammond, Liz O’Donovan, J. Hildyard, S. El Andaloussi, M. Gait, M. Wood, D. Wells   +13 more
semanticscholar   +1 more source