Your browser doesn't support javascript.
loading
"Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic.
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.
Afiliação
  • Gordish-Dressman H; Children's National Medical Center, Washington, DC, USA.
  • Willmann R; Biozentrum, University of Basel, Basel, Switzerland.
  • Dalle Pazze L; Charley's Fund, New York, NY, USA.
  • Kreibich A; Charley's Fund, New York, NY, USA.
  • van Putten M; Department of Human Genetics, Leiden University Medical Center, the Netherlands.
  • Heydemann A; Department of Physiology and Biophysics, University of Illinois at Chicago, Chicago, IL, USA.
  • Bogdanik L; The Jackson Laboratory, Bar Harbor, ME, USA.
  • Lutz C; The Jackson Laboratory, Bar Harbor, ME, USA.
  • Davies K; Department of Physiology, University of Oxford, Anatomy and Genetics, Oxford, UK.
  • Demonbreun AR; Center for Genetic Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
  • Duan D; Department of Molecular Microbiology and Immunology, Department of Neurology, Department of Biomedical Sciences and Department of Bioengineering, University of Missouri, Columbia, MO, USA.
  • Elsey D; Summit Therapeutics, Abingdon, Oxfordshire, UK.
  • Fukada SI; Laboratory of Molecular and Cellular Physiology, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka, Japan.
  • Girgenrath M; Rare Disease Research Unit, Pfizer, CA, USA.
  • Patrick Gonzalez J; Solid Biosciences, Cambridge, MA, USA.
  • Grounds MD; School of Human Science, the University of Western Australia, Perth, Australia.
  • Nichols A; Catabasis Pharmaceuticals, Cambridge, MA, USA.
  • Partridge T; Children's National Medical Center, Washington, DC, USA.
  • Passini M; Sarepta Therapeutics, Inc. Cambridge, MA, USA.
  • Sanarica F; Department of Pharmacy and Drug Sciences, Unit of Pharmacology, University of Bari "Aldo Moro", Italy.
  • Schnell FJ; Sarepta Therapeutics, Inc. Cambridge, MA, USA.
  • Wells DJ; Department of Comparative Biomedical Sciences, Royal Veterinary College, London, UK.
  • Yokota T; Department of Medical Genetics, University of Alberta, Canada.
  • Young CS; Department of Neurology, Molecular Biology Institute, Center for Duchenne Muscular Dystrophy at UCLA, University of California, Los Angeles, CA, USA.
  • Zhong Z; Wave Life Sciences, Cambridge, MA, USA.
  • Spurney C; Children's National Medical Center, Washington, DC, USA.
  • Spencer M; Department of Neurology, Molecular Biology Institute, Center for Duchenne Muscular Dystrophy at UCLA, University of California, Los Angeles, CA, USA.
  • De Luca A; Department of Pharmacy and Drug Sciences, Unit of Pharmacology, University of Bari "Aldo Moro", Italy.
  • Nagaraju K; School of Pharmacy and Pharmaceutical Sciences, Binghamton University, New York, USA.
  • Aartsma-Rus A; Department of Human Genetics, Leiden University Medical Center, the Netherlands.
J Neuromuscul Dis ; 5(4): 407-417, 2018.
Article em En | MEDLINE | ID: mdl-30198876
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.
Assuntos
Palavras-chave
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne / Modelos Animais de Doenças / Distrofia Muscular Animal Idioma: En Ano de publicação: 2018 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne / Modelos Animais de Doenças / Distrofia Muscular Animal Idioma: En Ano de publicação: 2018 Tipo de documento: Article