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“Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic: First Workshop Report: Examining current findings and opportunities around the emerging D2.B10-Dmd(mdx)/J (D2/mdx) model in context of the classic C57BL/10ScSn-Dmd(mdx)/J (Bl10/mdx)

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 modifier...

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Autores principales: Gordish-Dressman, Heather, Willmann, Raffaella, Dalle Pazze, Laura, Kreibich, Arati, van Putten, Maaike, Heydemann, Ahlke, Bogdanik, Laurent, Lutz, Cathleen, Davies, Kay, Demonbreun, Alexis R., Duan, Dongsheng, Elsey, David, Fukada, So-ichiro, Girgenrath, Mahasweta, Patrick Gonzalez, J., Grounds, Miranda D., Nichols, Andy, Partridge, Terry, Passini, Marco, Sanarica, Francesca, Schnell, Frederick J., Wells, Dominic J., Yokota, Toshifumi, Young, Courtney S., Zhong, Zhong, Spurney, Christopher, Spencer, Melissa, De Luca, Annamaria, Nagaraju, Kanneboyina, Aartsma-Rus, Annemieke
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6218134/
https://www.ncbi.nlm.nih.gov/pubmed/30198876
http://dx.doi.org/10.3233/JND-180324
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author Gordish-Dressman, Heather
Willmann, Raffaella
Dalle Pazze, Laura
Kreibich, Arati
van Putten, Maaike
Heydemann, Ahlke
Bogdanik, Laurent
Lutz, Cathleen
Davies, Kay
Demonbreun, Alexis R.
Duan, Dongsheng
Elsey, David
Fukada, So-ichiro
Girgenrath, Mahasweta
Patrick Gonzalez, J.
Grounds, Miranda D.
Nichols, Andy
Partridge, Terry
Passini, Marco
Sanarica, Francesca
Schnell, Frederick J.
Wells, Dominic J.
Yokota, Toshifumi
Young, Courtney S.
Zhong, Zhong
Spurney, Christopher
Spencer, Melissa
De Luca, Annamaria
Nagaraju, Kanneboyina
Aartsma-Rus, Annemieke
author_facet Gordish-Dressman, Heather
Willmann, Raffaella
Dalle Pazze, Laura
Kreibich, Arati
van Putten, Maaike
Heydemann, Ahlke
Bogdanik, Laurent
Lutz, Cathleen
Davies, Kay
Demonbreun, Alexis R.
Duan, Dongsheng
Elsey, David
Fukada, So-ichiro
Girgenrath, Mahasweta
Patrick Gonzalez, J.
Grounds, Miranda D.
Nichols, Andy
Partridge, Terry
Passini, Marco
Sanarica, Francesca
Schnell, Frederick J.
Wells, Dominic J.
Yokota, Toshifumi
Young, Courtney S.
Zhong, Zhong
Spurney, Christopher
Spencer, Melissa
De Luca, Annamaria
Nagaraju, Kanneboyina
Aartsma-Rus, Annemieke
author_sort Gordish-Dressman, Heather
collection PubMed
description 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.
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spelling pubmed-62181342018-11-07 “Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic: First Workshop Report: Examining current findings and opportunities around the emerging D2.B10-Dmd(mdx)/J (D2/mdx) model in context of the classic C57BL/10ScSn-Dmd(mdx)/J (Bl10/mdx) Gordish-Dressman, Heather Willmann, Raffaella Dalle Pazze, Laura Kreibich, Arati van Putten, Maaike Heydemann, Ahlke Bogdanik, Laurent Lutz, Cathleen Davies, Kay Demonbreun, Alexis R. Duan, Dongsheng Elsey, David Fukada, So-ichiro Girgenrath, Mahasweta Patrick Gonzalez, J. Grounds, Miranda D. Nichols, Andy Partridge, Terry Passini, Marco Sanarica, Francesca Schnell, Frederick J. Wells, Dominic J. Yokota, Toshifumi Young, Courtney S. Zhong, Zhong Spurney, Christopher Spencer, Melissa De Luca, Annamaria Nagaraju, Kanneboyina Aartsma-Rus, Annemieke J Neuromuscul Dis Review 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. IOS Press 2018-10-23 /pmc/articles/PMC6218134/ /pubmed/30198876 http://dx.doi.org/10.3233/JND-180324 Text en © 2018 – IOS Press and the authors. All rights reserved https://creativecommons.org/licenses/by-nc/4.0/ This is an open access article distributed under the terms of the Creative Commons Attribution Non-Commercial (CC BY-NC 4.0) License (https://creativecommons.org/licenses/by-nc/4.0/) , which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Review
Gordish-Dressman, Heather
Willmann, Raffaella
Dalle Pazze, Laura
Kreibich, Arati
van Putten, Maaike
Heydemann, Ahlke
Bogdanik, Laurent
Lutz, Cathleen
Davies, Kay
Demonbreun, Alexis R.
Duan, Dongsheng
Elsey, David
Fukada, So-ichiro
Girgenrath, Mahasweta
Patrick Gonzalez, J.
Grounds, Miranda D.
Nichols, Andy
Partridge, Terry
Passini, Marco
Sanarica, Francesca
Schnell, Frederick J.
Wells, Dominic J.
Yokota, Toshifumi
Young, Courtney S.
Zhong, Zhong
Spurney, Christopher
Spencer, Melissa
De Luca, Annamaria
Nagaraju, Kanneboyina
Aartsma-Rus, Annemieke
“Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic: First Workshop Report: Examining current findings and opportunities around the emerging D2.B10-Dmd(mdx)/J (D2/mdx) model in context of the classic C57BL/10ScSn-Dmd(mdx)/J (Bl10/mdx)
title “Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic: First Workshop Report: Examining current findings and opportunities around the emerging D2.B10-Dmd(mdx)/J (D2/mdx) model in context of the classic C57BL/10ScSn-Dmd(mdx)/J (Bl10/mdx)
title_full “Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic: First Workshop Report: Examining current findings and opportunities around the emerging D2.B10-Dmd(mdx)/J (D2/mdx) model in context of the classic C57BL/10ScSn-Dmd(mdx)/J (Bl10/mdx)
title_fullStr “Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic: First Workshop Report: Examining current findings and opportunities around the emerging D2.B10-Dmd(mdx)/J (D2/mdx) model in context of the classic C57BL/10ScSn-Dmd(mdx)/J (Bl10/mdx)
title_full_unstemmed “Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic: First Workshop Report: Examining current findings and opportunities around the emerging D2.B10-Dmd(mdx)/J (D2/mdx) model in context of the classic C57BL/10ScSn-Dmd(mdx)/J (Bl10/mdx)
title_short “Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic: First Workshop Report: Examining current findings and opportunities around the emerging D2.B10-Dmd(mdx)/J (D2/mdx) model in context of the classic C57BL/10ScSn-Dmd(mdx)/J (Bl10/mdx)
title_sort “of mice and measures”: a project to improve how we advance duchenne muscular dystrophy therapies to the clinic: first workshop report: examining current findings and opportunities around the emerging d2.b10-dmd(mdx)/j (d2/mdx) model in context of the classic c57bl/10scsn-dmd(mdx)/j (bl10/mdx)
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6218134/
https://www.ncbi.nlm.nih.gov/pubmed/30198876
http://dx.doi.org/10.3233/JND-180324
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