Tracking progress: an update on animal models for Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a progressive, fatal, X-linked monogenic muscle disorder caused by mutations in the gene. In order to test treatments for DMD, a range of natural and engineered animal models have been developed, including mice, rats, dogs and pigs. Sui and colleagues have now ad...

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Bibliographic Details
Published in:Disease models & mechanisms 2018-06, Vol.11 (6)
Main Author: Wells, Dominic J
Format: Article
Language:English
Subjects:
Animals
Cardiomyopathy
CRISPR-Cas Systems - genetics
Disease Models, Animal
Dog models
Duchenne muscular dystrophy
Genotype & phenotype
Hogs
Males
Mortality
Mouse models
Muscular dystrophy
Muscular Dystrophy, Duchenne - genetics
Muscular Dystrophy, Duchenne - pathology
Mutation
Mutation - genetics
Pathology
Pig models
Rabbit models
Rodents
Translational Medical Research
Ventilators
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Description
Summary:Duchenne muscular dystrophy (DMD) is a progressive, fatal, X-linked monogenic muscle disorder caused by mutations in the gene. In order to test treatments for DMD, a range of natural and engineered animal models have been developed, including mice, rats, dogs and pigs. Sui and colleagues have now added a dystrophic rabbit model to this range using CRISPR/Cas9 to disrupt exon 51 of Rabbits have the advantage of being easier to breed and less costly than dog or pig models, but having clear clinical signs, in contrast to many mouse models. There appears to be an effect of body size in models of DMD, as the severity of the clinical signs increases with increasing body size across species. All DMD models have advantages and disadvantages, and it is crucial that investigators understand the limitations of each model when testing novel therapies for DMD in pre-clinical studies.
ISSN:1754-8403
1754-8411
DOI:10.1242/dmm.035774