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Keywords: Disease model
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Journal Articles
Dis Model Mech (2021) 14 (2): dmm047548.
Published: 11 February 2021
... lateral sclerosis and frontotemporal dementia, using the eye as a model and demonstrated axonal cytoskeleton alterations. TDP-43 Amyotrophic lateral sclerosis Frontotemporal lobar degeneration Neurodegeneration Disease model Visual system Amyotrophic lateral sclerosis (ALS) and...
Includes: Supplementary data
Journal Articles
Dis Model Mech (2019) 12 (12): dmm041368.
Published: 19 December 2019
... available models, and discuss emerging modeling technologies. Core myopathy Disease model Skeletal muscle The core myopathies are the most prevalent subgroup of congenital myopathies ( Amburgey et al., 2011 ; Gonorazky et al., 2018 ; Maggi et al., 2013 ; Norwood et al., 2009 ), comprising...
Journal Articles
Dis Model Mech (2019) 12 (11): dmm040576.
Published: 04 November 2019
... repurposing screens and identified the FDA-approved drug aripiprazole as a clinical candidate. N -glycanase 1 deficiency NGLY1 SKN-1 Pngl Congenital disorder of deglycosylation Disease model Aripiprazole NGLY1 deficiency was the first congenital disorder of deglycosylation (CDDG...
Includes: Supplementary data
Journal Articles
Dis Model Mech (2019) 12 (2): dmm039271.
Published: 22 February 2019
... interventions for rare diseases. In this Editorial, we point to the key parameters in face, construct, predictive and target validity for accurate disease modelling, with special emphasis on rare disease models. Raising the experimental standards for disease models will enhance successful clinical translation...
Journal Articles
Dis Model Mech (2018) 11 (3): dmm032482.
Published: 27 March 2018
... ignored in animal models. The impact of this finding, and an approach to overcome it, will be discussed in the context of interpreting disease model characterization and intervention studies. This article has an associated First Person interview with the first author of the paper. * Authors for...
Includes: Supplementary data
Journal Articles
In collection:
Stem Cells
Dis Model Mech (2017) 10 (9): 1039–1059.
Published: 01 September 2017
... resulted in a paradigm shift in cardiac disease modelling. Although hPSCs are unlikely to completely replace animal or heterologous cell-based model systems, hPSC-CMs have proven to be a powerful platform to model various cardiac disorders. This has led to novel mechanistic insights into disease...
Journal Articles
Dis Model Mech (2016) 9 (3): 245–252.
Published: 01 March 2016
... disease models in recent years. In this article, we highlight recent efforts to improve the availability and accessibility of the disease model information in FlyBase (http://flybase.org), the model organism database for Drosophila . FlyBase has recently introduced Human Disease Model Reports, each of...
Journal Articles
Dis Model Mech (2014) 7 (7): 769–776.
Published: 01 July 2014
... Zebrafish Disease model Gastrointestinal Hematovascular Regenerative medicine offers the promise of regaining organ function after acute or chronic injury. Regenerative approaches aim to promote, enhance and re-establish organ-specific repair processes to reconstitute organ structure and function...