Results 1 to 10 of about 59,671 (341)

Lentiviral vectors can be used for full-length dystrophin gene therapy [PDF]

Scientific Reports, 2017
Duchenne Muscular Dystrophy (DMD) is caused by a lack of dystrophin expression in patient muscle fibres. Current DMD gene therapy strategies rely on the expression of internally deleted forms of dystrophin, missing important functional domains.
John R. Counsell, Zeinab Asgarian, Jinhong Meng, Veronica Ferrer, Conrad A. Vink, Steven J. Howe, Simon N. Waddington, Adrian J. Thrasher, Francesco Muntoni, Jennifer E. Morgan, Olivier Danos   +10 more
doaj   +5 more sources

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

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

Nature Communications, 2017
Gene replacement therapies utilizing adeno-associated viral (AAV) vectors hold great promise for treating Duchenne muscular dystrophy (DMD). A related approach uses AAV vectors to edit specific regions of the DMD gene using CRISPR/Cas9.
Guy L Odom, Michael P Phelps, R David Hawkins   +2 more
exaly   +2 more sources

In vivo non-invasive monitoring of dystrophin correction in a new Duchenne muscular dystrophy reporter mouse

Nature Communications, 2019
Dystrophin-deficient mice are used to test corrective strategies for Duchenne muscular dystrophy, but evaluation of dystrophin expression requires collection of tissue samples from specific muscles and time points.
Leonela Amoasii, Hui Li, Yu Zhang, Yi-Li Min, Efrain Sanchez-Ortiz, John M. Shelton, Chengzu Long, Alex A. Mireault, Samadrita Bhattacharyya, John R. McAnally, Rhonda Bassel-Duby, Eric N. Olson   +11 more
doaj   +2 more sources

Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division

Nature Medicine, 2015
Dystrophin is expressed in differentiated myofibers, in which it is required for sarcolemmal integrity, and loss-of-function mutations in the gene that encodes it result in Duchenne muscular dystrophy (DMD), a disease characterized by progressive and ...
Nicolas A Dumont, Yu Xin Wang, Julia von Maltzahn   +2 more
exaly   +2 more sources

Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy

Nature Communications, 2015
The CRISPR/Cas9 genome-editing platform is a promising technology to correct the genetic basis of hereditary diseases. The versatility, efficiency and multiplexing capabilities of the CRISPR/Cas9 system enable a variety of otherwise challenging gene ...
Pratiksha I Thakore, William H Majoros, Timothy E Reddy   +2 more
exaly   +2 more sources

Necroptosis mediates myofibre death in dystrophin-deficient mice

Nature Communications, 2018
Duchenne muscular dystrophy (DMD) is a severe degenerative disorder caused by mutations in the dystrophin gene. Dystrophin-deficient muscles are characterised by progressive myofibre necrosis in which inflammation plays a deleterious role.
Alexandre Prola, Virginie Mariot, Julie Dumonceaux   +2 more
exaly   +2 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

Dystrophin As a Molecular Shock Absorber

ACS Nano, 2018
Dystrophin is the largest protein isoform (427 kDa) expressed from the gene defective in Duchenne muscular dystrophy, a lethal muscle-wasting and genetically inherited disease.
Shimin Le, Miao Yu, Ladislav Hovan
exaly   +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