Predictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophy
| Autor(a) principal: | |
|---|---|
| Data de Publicação: | 2014 |
| Outros Autores: | , , , , , , , , , |
| Tipo de documento: | Artigo |
| Idioma: | eng |
| Título da fonte: | Repositório Institucional da FIOCRUZ (ARCA) |
| Texto Completo: | https://www.arca.fiocruz.br/handle/icict/10676 |
Resumo: | Université Paris-Est. Ecole Nationale Vétérinaire d’Alfort. UPR de Neurobiologie. Paris, France. |
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Barthélémy, InêsMariz, Fernanda PintoYada, EricaDesquilbet, LoïcSavino, WilsonBarbosa, Suse Dayse SilvaFaussat, Anne-MarieMouly, VincentVoit, ThomasBlot, StéphaneButler-Browne, Gillian2015-06-08T14:01:46Z2015-06-08T14:01:46Z2014BARTHÉLÉMY, Inès et al. Predictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophy. Disease Models & Mechanisms, v.7, n.11, p.1253-1261, 2014.1754-8403https://www.arca.fiocruz.br/handle/icict/1067610.1242/dmm.016014engThe Company of Biologists LtdLinfócitosGRMDDMDDistrophinPredictive biomakerLymphocyteCD49dGait analysisAccelerometryAcelerometriaDystrophinPredictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophyinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleUniversité Paris-Est. Ecole Nationale Vétérinaire d’Alfort. UPR de Neurobiologie. Paris, France.Université Pierre et Marie Curie-Paris. Institut de Myologie. Paris, France/ Universidade Federal do Rio de Janeiro. Instituto de Pediatria. Rio de Janeiro, RJ, Brasil / Fundação Oswaldo Cruz. Instituto Oswaldo Cruz. Laboratório de Pesquisa sobre o Timo. Rio de Janeiro, RJ, Brasil.Université Pierre et Marie Curie-Paris . Institut de Myologie. Paris, France.Université Paris-Est. Ecole Nationale Vétérinaire d’Alfort. Unité D’Épidémiologie clinique et de Biostatistique. Paris, France / CNRS UMR 7179, MNHN, Brunoy 91800, France.Fundação Oswaldo Cruz. Instituto Oswaldo Cruz. Laboratório de Pesquisa sobre o Timo. Rio de Janeiro, RJ, Brasil.Instituto Nacional de Câncer (INCA). Departamento de Pesquisa Clínica. Rio de Janeiro, RJ, Brasil.Université Pierre et Marie Curie-Paris . Institut de Myologie. Paris, France.Université Pierre et Marie Curie-Paris . Institut de Myologie. Paris, France.Université Paris-Est. Ecole Nationale Vétérinaire d’Alfort. UPR de Neurobiologie. Paris, France.Université Pierre et Marie Curie-Paris . Institut de Myologie. Paris, France.In the translational process of developing innovative therapies for DMD (Duchenne muscular dystrophy), the last preclinical validation step is often carried out in the most relevant animal model of this human disease, namely the GRMD (Golden Retriever muscular dystrophy) dog. The disease in GRMD dogs mimics human DMD in many aspects, including the inter-individual heterogeneity. This last point can be seen as a drawback for an animal model but is inherently related to the disease in GRMD dogs closely resembling that of individuals with DMD. In order to improve the management of this inter-individual heterogeneity, we have screened a combination of biomarkers in sixty-one 2-month-old GRMD dogs at the onset of the disease and a posteriori we addressed their predictive value on the severity of the disease. Three non-invasive biomarkers obtained at early stages of the disease were found to be highly predictive for the loss of ambulation before 6 months of age. An elevation in the number of circulating CD4+CD49dhi T cells and a decreased stride frequency resulting in a reduced spontaneous speed were found to be strongly associated with the severe clinical form of the disease. These factors can be used as predictive tests to screen dogs to separate them into groups with slow or fast disease progression before their inclusion into a therapeutic preclinical trial, and therefore improve the reliability and translational value of the trials carried out on this invaluable large animal model. These same biomarkers have also been described to be predictive for the time to loss of ambulation in boys with DMD, strengthening the relevance of GRMD dogs as preclinical models of this devastating muscle disease.info:eu-repo/semantics/openAccessreponame:Repositório Institucional da FIOCRUZ (ARCA)instname:Fundação Oswaldo Cruz (FIOCRUZ)instacron:FIOCRUZLICENSElicense.txttext/plain1914https://www.arca.fiocruz.br/bitstream/icict/10676/1/license.txt7d48279ffeed55da8dfe2f8e81f3b81fMD51ORIGINALfernanda_marizetal_IOC_2014.pdfapplication/pdf928634https://www.arca.fiocruz.br/bitstream/icict/10676/2/fernanda_marizetal_IOC_2014.pdfbea5c3f8ed6d81901b351d7c4243f0b8MD52TEXTfernanda_marizetal_IOC_2014.pdf.txtfernanda_marizetal_IOC_2014.pdf.txtExtracted texttext/plain51342https://www.arca.fiocruz.br/bitstream/icict/10676/3/fernanda_marizetal_IOC_2014.pdf.txt665a827bb53e7d2858f03c801d1e871cMD53icict/106762022-06-24 12:17:38.162oai:www.arca.fiocruz.br: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ório InstitucionalPUBhttps://www.arca.fiocruz.br/oai/requestrepositorio.arca@fiocruz.bropendoar:21352022-06-24T15:17:38Repositório Institucional da FIOCRUZ (ARCA) - Fundação Oswaldo Cruz (FIOCRUZ)false |
| dc.title.pt_BR.fl_str_mv |
Predictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophy |
| title |
Predictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophy |
| spellingShingle |
Predictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophy Barthélémy, Inês Linfócitos GRMD DMD Distrophin Predictive biomaker Lymphocyte CD49d Gait analysis Accelerometry Acelerometria Dystrophin |
| title_short |
Predictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophy |
| title_full |
Predictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophy |
| title_fullStr |
Predictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophy |
| title_full_unstemmed |
Predictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophy |
| title_sort |
Predictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophy |
| author |
Barthélémy, Inês |
| author_facet |
Barthélémy, Inês Mariz, Fernanda Pinto Yada, Erica Desquilbet, Loïc Savino, Wilson Barbosa, Suse Dayse Silva Faussat, Anne-Marie Mouly, Vincent Voit, Thomas Blot, Stéphane Butler-Browne, Gillian |
| author_role |
author |
| author2 |
Mariz, Fernanda Pinto Yada, Erica Desquilbet, Loïc Savino, Wilson Barbosa, Suse Dayse Silva Faussat, Anne-Marie Mouly, Vincent Voit, Thomas Blot, Stéphane Butler-Browne, Gillian |
| author2_role |
author author author author author author author author author author |
| dc.contributor.author.fl_str_mv |
Barthélémy, Inês Mariz, Fernanda Pinto Yada, Erica Desquilbet, Loïc Savino, Wilson Barbosa, Suse Dayse Silva Faussat, Anne-Marie Mouly, Vincent Voit, Thomas Blot, Stéphane Butler-Browne, Gillian |
| dc.subject.other.pt_BR.fl_str_mv |
Linfócitos |
| topic |
Linfócitos GRMD DMD Distrophin Predictive biomaker Lymphocyte CD49d Gait analysis Accelerometry Acelerometria Dystrophin |
| dc.subject.en.pt_BR.fl_str_mv |
GRMD DMD Distrophin Predictive biomaker Lymphocyte CD49d Gait analysis Accelerometry |
| dc.subject.decs.pt_BR.fl_str_mv |
Acelerometria Dystrophin |
| description |
Université Paris-Est. Ecole Nationale Vétérinaire d’Alfort. UPR de Neurobiologie. Paris, France. |
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2014 |
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2014 |
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2015-06-08T14:01:46Z |
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2015-06-08T14:01:46Z |
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info:eu-repo/semantics/publishedVersion |
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info:eu-repo/semantics/article |
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BARTHÉLÉMY, Inès et al. Predictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophy. Disease Models & Mechanisms, v.7, n.11, p.1253-1261, 2014. |
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https://www.arca.fiocruz.br/handle/icict/10676 |
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1754-8403 |
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10.1242/dmm.016014 |
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BARTHÉLÉMY, Inès et al. Predictive markers of clinical outcome in the GRMD dog model of Duchenne muscular dystrophy. Disease Models & Mechanisms, v.7, n.11, p.1253-1261, 2014. 1754-8403 10.1242/dmm.016014 |
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