Results 251 to 260 of about 59,671 (341)

Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine Model.

Human Gene Therapy, 2017
Kasun Kodippili, Chady H. Hakim, Xiufang Pan, Hsiao T. Yang, Y. Yue, Yadong Zhang, Jin-Hong Shin, N. N. Yang, D. Duan   +8 more
semanticscholar   +1 more source

Enhanced therapeutic potential of a microdystrophin with an extended C-terminal domain. [PDF]

Mol Ther Methods Clin Dev
Foltz SJ, Qian R, Yang J, Elliott K, Heithoff BP, Jasick H, Smith JB, Karumuthil-Melethil S, Qiao C, Liu Y, Danos O.   +10 more
europepmc   +1 more source

Association of Aciculin with Dystrophin and Utrophin

, 1995
Alexey M. Belkin, Keith Burridge
openalex   +2 more sources

Friend or Foe?: The Immune System's Dilemma With Dystrophin Replacement Therapy. [PDF]

JACC Basic Transl Sci
Townsend D.
europepmc   +1 more source

Fibrillin-Related Proteins Control Calcium Homeostasis in Dystrophic Muscle Across Species. [PDF]

MicroPubl Biol
Marchiafava D, Vidal Gadea A.
europepmc   +1 more source

Central Neurophysiological Alterations in Dystrophic mdx Mice Correlate With Reduced Hippocampal Levels of the Endogenous NMDA Receptor Ligand D-Aspartate. [PDF]

J Neurochem
Mastrostefano F, Garofalo M, Nuzzo T, Bruno C, Errico F, Usiello A, De Stefano ME.   +6 more
europepmc   +1 more source

Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy

Human Genetics, 2016
J. Robinson-Hamm, C. Gersbach
semanticscholar   +1 more source

AAV mini-dystrophin gene therapy for Duchenne muscular dystrophy: a phase 1b trial. [PDF]

Nat Med
Butterfield RJ, Shieh PB, Li H, Binks M, McDonnell TG, Ryan KA, Delnomdedieu M, Belluscio BA, Neelakantan S, Levy DI, Schwartz PF, Smith EC.   +11 more
europepmc   +1 more source

Muscle-specific gene editing therapy via mammalian fusogen-directed virus-like particles. [PDF]

Nat Commun
Zhou SK, Luo JT, Chen YF, Lu ZD, Jian QH, Jiang SQ, Li J, Zhang XQ, Tan XY, Yang XZ, Xu CF, Wang J.   +11 more
europepmc   +1 more source

Age-related Differences in Dystrophin: Impact on Force Transfer Proteins, Membrane Integrity, and Neuromuscular Junction Stability

The journals of gerontology. Series A, Biological sciences and medical sciences, 2016
David C. Hughes, George R. Marcotte, A. Marshall, Daniel W D West, Leslie M. Baehr, M. Wallace, Perrie M Saleh, S. Bodine, K. Baar   +8 more
semanticscholar   +1 more source