Tracking Progress: an Update on Animal Models for Duchenne Muscular Dystrophy

Overview

Journal Dis Model Mech

Specialty General Medicine

Date 2018 Jun 20

PMID 29914884

Citations 28

Authors

Dominic J Wells

Affiliations

Soon will be listed here.

Abstract

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.

Citing Articles

Changes to the Autophagy-Related Muscle Proteome Following Short-Term Treatment with Ectoine in the Duchenne Muscular Dystrophy Mouse Model mdx.

Gomez Armengol E, Merckx C, De Sutter H, De Bleecker J, De Paepe B Int J Mol Sci. 2025; 26(2).

PMID: 39859157 PMC: 11765399. DOI: 10.3390/ijms26020439.

Identification of reference microRNAs in skeletal muscle of a canine model of Duchenne muscular dystrophy.

Riddell D, Hildyard J, Harron R, Wells D, Piercy R Wellcome Open Res. 2024; 9:362.

PMID: 39649621 PMC: 11621615. DOI: 10.12688/wellcomeopenres.22481.2.

Longitudinal assessment of skeletal muscle functional mechanics in the DE50-MD dog model of Duchenne muscular dystrophy.

Riddell D, Hildyard J, Harron R, Taylor-Brown F, Kornegay J, Wells D Dis Model Mech. 2023; 16(12).

PMID: 38050706 PMC: 10753191. DOI: 10.1242/dmm.050395.

Identification of quantitative polymerase chain reaction reference genes suitable for normalising gene expression in the brain of normal and dystrophic mice and dogs.

Crawford A, Hildyard J, Wells D, Piercy R Wellcome Open Res. 2023; 6:84.

PMID: 37942409 PMC: 10628364. DOI: 10.12688/wellcomeopenres.16707.2.

Receptor interacting protein kinase-3 mediates both myopathy and cardiomyopathy in preclinical animal models of Duchenne muscular dystrophy.

Bencze M, Periou B, Punzon I, Barthelemy I, Taglietti V, Hou C J Cachexia Sarcopenia Muscle. 2023; 14(6):2520-2531.

PMID: 37909859 PMC: 10751447. DOI: 10.1002/jcsm.13265.

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