Cargando…
“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...
Autores principales: | , , , , , , , , , , , , , , , , , , , , , , , , , , , , , |
---|---|
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 |
_version_ | 1783368410047971328 |
---|---|
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. |
format | Online Article Text |
id | pubmed-6218134 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2018 |
publisher | IOS Press |
record_format | MEDLINE/PubMed |
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 |
work_keys_str_mv | AT gordishdressmanheather ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT willmannraffaella ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT dallepazzelaura ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT kreibicharati ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT vanputtenmaaike ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT heydemannahlke ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT bogdaniklaurent ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT lutzcathleen ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT davieskay ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT demonbreunalexisr ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT duandongsheng ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT elseydavid ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT fukadasoichiro ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT girgenrathmahasweta ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT patrickgonzalezj ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT groundsmirandad ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT nicholsandy ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT partridgeterry ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT passinimarco ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT sanaricafrancesca ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT schnellfrederickj ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT wellsdominicj ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT yokotatoshifumi ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT youngcourtneys ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT zhongzhong ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT spurneychristopher ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT spencermelissa ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT delucaannamaria ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT nagarajukanneboyina ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx AT aartsmarusannemieke ofmiceandmeasuresaprojecttoimprovehowweadvanceduchennemusculardystrophytherapiestotheclinicfirstworkshopreportexaminingcurrentfindingsandopportunitiesaroundtheemergingd2b10dmdmdxjd2mdxmodelincontextoftheclassicc57bl10scsndmdmdxjbl10mdx |