scholarly journals Sustained AAV-mediated Dystrophin Expression in a Canine Model of Duchenne Muscular Dystrophy with a Brief Course of Immunosuppression

2007 ◽  
Vol 15 (6) ◽  
pp. 1160-1166 ◽  
Author(s):  
Zejing Wang ◽  
Christian S Kuhr ◽  
James M Allen ◽  
Michael Blankinship ◽  
Paul Gregorevic ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Canine Model ◽  
Dystrophin Expression

scholarly journals Validation of DE50-MD dogs as a model for the brain phenotype of Duchenne muscular dystrophy

2022 ◽  
Author(s):  
Abbe H. Crawford ◽  
John C.W. Hildyard ◽  
Sophie A.M. Rushing ◽  
Dominic J. Wells ◽  
Maria Diez-Leon ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Canine Model ◽  
Clinical Model ◽  
Dystrophin Expression ◽  
Canine Brain ◽  
Dystrophin Deficiency ◽  
Novel Objects ◽  
And Function ◽  
The Brain

Duchenne muscular dystrophy (DMD), a fatal musculoskeletal disorder, is associated with neurodevelopmental disorders and cognitive impairment caused by brain dystrophin deficiency. Dog models of DMD represent key translational tools to study dystrophin biology and to develop novel therapeutics. However, characterization of dystrophin expression and function in the canine brain is lacking. We studied the DE50-MD canine model of DMD that has a missense mutation in the donor splice site of exon 50. Using a battery of cognitive tests, we detected a neurocognitive phenotype in DE50-MD dogs including reduced attention, problem-solving and exploration of novel objects. Through a combination of capillary immunoelectrophoresis, immunolabelling, qPCR and RNAScope in situ hybridization we show that regional dystrophin expression in the adult canine brain reflects that of humans, and that the DE50-MD dog lacks full length dystrophin (Dp427) protein expression but retains expression of the two shorter brain-expressed isoforms, Dp140 and Dp71. Thus, the DE50-MD dog is a translationally-relevant pre-clinical model to study the consequences of Dp427 deficiency in the brain and to develop therapeutic strategies for the neurological sequelae of DMD.

MRI-based quantification of Duchenne muscular dystrophy in a canine model

2011 ◽  
Author(s):  
Jiahui Wang ◽  
Zheng Fan ◽  
Joe N. Kornegay ◽  
Martin A. Styner
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Canine Model

scholarly journals Restoring Dystrophin Expression in Duchenne Muscular Dystrophy Muscle

2011 ◽  
Vol 179 (1) ◽  
pp. 12-22 ◽  
Author(s):  
Eric P. Hoffman ◽  
Abby Bronson ◽  
Arthur A. Levin ◽  
Shin'ichi Takeda ◽  
Toshifumi Yokota ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Dystrophin Expression

scholarly journals Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger Nucleases

2015 ◽  
Vol 23 (3) ◽  
pp. 523-532 ◽  
Author(s):  
David G Ousterout ◽  
Ami M Kabadi ◽  
Pratiksha I Thakore ◽  
Pablo Perez-Pinera ◽  
Matthew T Brown ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Zinc Finger ◽  
Zinc Finger Nucleases ◽  
Dystrophin Expression ◽  
In Cells
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