Results 141 to 150 of about 56,717 (273)

Targeting a therapeutic LIF transgene to muscle via the immune system ameliorates muscular dystrophy. [PDF]

, 2019
Many potentially therapeutic molecules have been identified for treating Duchenne muscular dystrophy. However, targeting those molecules only to sites of active pathology is an obstacle to their clinical use.
Bertoni, Carmen, Flores, Ivan, Ramos, Julian, Tidball, James G, Wang, Ying, Wehling-Henricks, Michelle, Welc, Steven S   +6 more
core  

Utrophin Compensates dystrophin Loss during Mouse Spermatogenesis

Scientific Reports, 2017
Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder resulting from mutations in the dystrophin gene. The mdx/utrn −/− mouse, lacking in both dystrophin and its autosomal homologue utrophin, is commonly used to model the clinical symptoms of
Hung-Chih Chen, Yu-Feng Chin, David J. Lundy, Chung-Tiang Liang, Ya-Hui Chi, Paolin Kuo, Patrick C. H. Hsieh   +6 more
doaj   +1 more source

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

Science, 2016
Chengzu Long, L. Amoasii, A. Mireault, John R. McAnally, Hui Li, E. Sánchez-Ortiz, Samadrita Bhattacharyya, J. Shelton, R. Bassel-Duby, E. Olson   +9 more
semanticscholar   +1 more source

Absence of dystrophin alters the passive properties of the extensor digitorum longus muscle in mice [PDF]

, 2010
Dystrophin is a cytoskeletal protein not directly participating the myosin-actin contractile apparatus in muscle. The loss of dystrophin leads to Duchenne muscular dystrophy. It is well-established that contractility is reduced in dystrophin-null muscle.
Hakim, Chady H.
core  

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

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   +1 more source

Dystrophin expression in the mdx mouse restored by stem cell transplantation

Nature, 1999
E. Gussoni, Y. Soneoka, Corinne D. Strickland, E. Buzney, Mohamed K Khan, A. Flint, L. Kunkel, R. Mulligan   +7 more
semanticscholar   +1 more source

Dystrophin [PDF]

, 2020
openaire   +1 more source

Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy

Scientific Reports
Delandistrogene moxeparvovec is an rAAVrh74 vector-based gene transfer therapy that delivers a transgene encoding delandistrogene moxeparvovec micro-dystrophin, an engineered, functional form of dystrophin shown to stabilize or slow disease progression ...
Rachael A. Potter, Ida H. Moeller, Sohrab Khan, Hélène Haegel, Andreas Hollenstein, Guido Steiner, Christoph Wandel, Alexander P. Murphy, Damon R. Asher, Emanuel Palatinsky, Danielle A. Griffin, Stefanie Mason, Susan T. Iannaccone, Craig M. Zaidman, Louise R. Rodino-Klapac   +14 more
doaj   +1 more source

Gettysburg: Our College\u27s Magazine Winter 2018

, 2018
From the President Janet Morgan Riggs \u2777 Table of Contents Virtual Dissection Tables to Enhance Study of Anatomy Prof Notes: Jennifer Bloomquist (Professor Jennifer Bloomquist) Life\u27s Lessons Learned from Sports & Science (Sarah Hansen \u2717 ...
Communications & Marketing,
core   +1 more source

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

Science Translational Medicine, 2017
L. Amoasii, Chengzu Long, Hui Li, A. Mireault, J. Shelton, E. Sánchez-Ortiz, John R. McAnally, Samadrita Bhattacharyya, F. Schmidt, D. Grimm, S. Hauschka, R. Bassel-Duby, E. Olson   +12 more
semanticscholar   +1 more source