Document Type
Article
Publication Date
1-1-2018
JAX Source
J Neuromuscul Dis 2018; 5(4):407-417
Volume
5
Issue
4
First Page
407
Last Page
417
ISSN
2214-3599
PMID
30198876
DOI
https://doi.org/10.3233/JND-180324
Abstract
A new line of dystrophic mdx mice on the DBA/2J (D2) background has emerged as a candidate to study the efficacy of therapeutic approaches for Duchenne muscular dystrophy (DMD). These mice harbor genetic polymorphisms that appear to increase the severity of the dystropathology, with disease modifiers that also occur in DMD patients, making them attractive for efficacy studies and drug development. This workshop aimed at collecting and consolidating available data on the pathological features and the natural history of these new D2/mdx mice, for comparison with classic mdx mice and controls, and to identify gaps in information and their potential value. The overall aim is to establish guidance on how to best use the D2/mdx mouse model in preclinical studies.
Recommended Citation
Gordish-Dressman H,
Willmann R,
Dalle Pazze L,
Kreibich A,
van Putten M,
Heydemann A,
Bogdanik LP,
Lutz C,
Davies K,
Demonbruen A,
Duan D,
Elsey D,
Fukada S,
Girgenrath M,
Patrick Gonzalez J,
Grounds M,
Nichols A,
Partridge T,
Passini M,
Sanarica F,
Schnell F,
Wells D,
Yokota T,
Young C,
Zhong Z,
Spurney C,
Spencer M,
De Luca A,
Nagaraju K,
Aartsma-Rus A.
"Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic. J Neuromuscul Dis 2018; 5(4):407-417
Comments
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