scholarly journals Respiratory Pathology in a Humanized Mouse Model of Duchenne Muscular Dystrophy

2021 ◽  
Vol 35 (S1) ◽  
Author(s):  
Angela McCall ◽  
Aidan Bailey ◽  
Logan Pucci ◽  
Justin Dhindsa ◽  
Jacqueline Robinson‐Hamm ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Mouse Model ◽  
Humanized Mouse ◽  
Humanized Mouse Model ◽  
Respiratory Pathology

scholarly journals Full-length Dystrophin Restoration via Targeted Genomic Integration by AAV-CRISPR in a Humanized Mouse Model of Duchenne Muscular Dystrophy

2021 ◽  
Author(s):  
Adrian Pickar-Oliver ◽  
Veronica Gough ◽  
Joel D. Bohning ◽  
Siyan Liu ◽  
Jacqueline N. Robinson-Hamm ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Mouse Model ◽  
Full Length ◽  
Humanized Mouse ◽  
Humanized Mouse Model ◽  
Genomic Integration

scholarly journals Preclinical platform for long-term evaluation of immuno-oncology drugs using hCD34+ humanized mouse model

2020 ◽  
Vol 8 (2) ◽  
pp. e001513
Author(s):  
Nahee Park ◽  
Kamal Pandey ◽  
Sei Kyung Chang ◽  
Ah-Young Kwon ◽  
Young Bin Cho ◽  
...  
Keyword(s):  
Mouse Model ◽  
Humanized Mice ◽  
Preclinical Models ◽  
Humanized Mouse ◽  
Humanized Mouse Model ◽  
Durable Response ◽  
Term Efficacy ◽  
Nsg Mice ◽  
Oncology Research

BackgroundWell-characterized preclinical models are essential for immune-oncology research. We investigated the feasibility of our humanized mouse model for evaluating the long-term efficacy of immunotherapy and biomarkers.MethodsHumanized mice were generated by injecting human fetal cord blood-derived CD34+ hematopoietic stem cells to NOD-scid IL2rγnull (NSG) mice myeloablated with irradiation or busulfan. The humanization success was defined as a 25% or higher ratio of human CD45+ cells to mice peripheral blood mononuclear cells.ResultsBusulfan was ultimately selected as the appropriate myeloablative method because it provided a higher success rate of humanization (approximately 80%) and longer survival time (45 weeks). We proved the development of functional T cells by demonstrating the anticancer effect of the programmed cell death-1 (PD-1) inhibitor in our humanized mice but not in non-humanized NSG mice. After confirming the long-lasting humanization state (45 weeks), we further investigated the response durability of the PD-1 inhibitor and biomarkers in our humanized mice. Early increase in serum tumor necrosis factor α levels, late increase in serum interleukin 6 levels and increase in tumor-infiltrating CD8+ T lymphocytes correlated more with a durable response over 60 days than with a non-durable response.ConclusionsOur CD34+ humanized mouse model is the first in vivo platform for testing the long-term efficacy of anticancer immunotherapies and biomarkers, given that none of the preclinical models has ever been evaluated for such a long duration.

scholarly journals TENASCIN C PROMOTES ENDOTHELIAL DYSFUNCTION ACCOMPANIED WITH CARDIAC FIBROSIS IN A MOUSE MODEL OF DUCHENNE MUSCULAR DYSTROPHY

2021 ◽  
Vol 77 (18) ◽  
pp. 841
Author(s):  
Petra Lujza Szabo ◽  
Ouafa Hamza ◽  
Milat Inci ◽  
Janine Ebner ◽  
Karlheinz Hilber ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Endothelial Dysfunction ◽  
Muscular Dystrophy ◽  
Mouse Model ◽  
Cardiac Fibrosis ◽  
Tenascin C
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