TY - JOUR
T1 - "of mice and measures"
T2 - A project to improve how we advance duchenne muscular dystrophy therapies to the clinic
AU - Gordish-Dressman, Heather
AU - Willmann, Raffaella
AU - Dalle Pazze, Laura
AU - Kreibich, Arati
AU - Van Putten, Maaike
AU - Heydemann, Ahlke
AU - Bogdanik, Laurent
AU - Lutz, Cathleen
AU - Davies, Kay
AU - Demonbruen, Alexis R.
AU - Duan, Dongsheng
AU - Elsey, David
AU - Fukada, So Ichiro
AU - Girgenrath, Mahasweta
AU - Patrick Gonzalez, J.
AU - Grounds, Miranda D.
AU - Nichols, Andy
AU - Partridge, Terry
AU - Passini, Marco
AU - Sanarica, Francesca
AU - Schnell, Frederick J.
AU - Wells, Dominic J.
AU - Yokota, Toshifumi
AU - Young, Courtney S.
AU - Zhong, Zhong
AU - Spurney, Christopher
AU - Spencer, Melissa
AU - De Luca, Annamaria
AU - Nagaraju, Kanneboyina
AU - Aartsma-Rus, Annemieke
PY - 2018/1/1
Y1 - 2018/1/1
N2 - 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.
AB - 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.
KW - DBA/2J
KW - DMD
KW - Duchenne muscular dystrophy
KW - mdx
UR - http://www.scopus.com/inward/record.url?scp=85055613653&partnerID=8YFLogxK
U2 - 10.3233/JND-180324
DO - 10.3233/JND-180324
M3 - Review article
C2 - 30198876
AN - SCOPUS:85055613653
SN - 2214-3599
VL - 5
SP - 407
EP - 417
JO - Journal of Neuromuscular Diseases
JF - Journal of Neuromuscular Diseases
IS - 4
ER -