"of mice and measures": A project to improve how we advance duchenne muscular dystrophy therapies to the clinic

Heather Gordish-Dressman, Raffaella Willmann, Laura Dalle Pazze, Arati Kreibich, Maaike Van Putten, Ahlke Heydemann, Laurent Bogdanik, Cathleen Lutz, Kay Davies, Alexis R. Demonbruen, Dongsheng Duan, David Elsey, So Ichiro Fukada, Mahasweta Girgenrath, J. Patrick Gonzalez, Miranda D. Grounds, Andy Nichols, Terry Partridge, Marco Passini, Francesca SanaricaFrederick J. Schnell, Dominic J. Wells, Toshifumi Yokota, Courtney S. Young, Zhong Zhong, Christopher Spurney, Melissa Spencer, Annamaria De Luca, Kanneboyina Nagaraju, Annemieke Aartsma-Rus

Research output: Contribution to journalReview article

9 Citations (Scopus)

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.

Original languageEnglish
Pages (from-to)407-417
Number of pages11
JournalJournal of Neuromuscular Diseases
Volume5
Issue number4
DOIs
Publication statusPublished - 1 Jan 2018

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    Gordish-Dressman, H., Willmann, R., Dalle Pazze, L., Kreibich, A., Van Putten, M., Heydemann, A., Bogdanik, L., Lutz, C., Davies, K., Demonbruen, A. R., Duan, D., Elsey, D., Fukada, S. I., Girgenrath, M., Patrick Gonzalez, J., Grounds, M. D., Nichols, A., Partridge, T., Passini, M., ... Aartsma-Rus, A. (2018). "of mice and measures": A project to improve how we advance duchenne muscular dystrophy therapies to the clinic. Journal of Neuromuscular Diseases, 5(4), 407-417. https://doi.org/10.3233/JND-180324