In vitro models to study the molecular mechanisms altered in motor neuron and NMJ diseases.
In vitro models that mimic the pathophysiological alterations observed in patients and animal models of neurodegenerative diseases are fundamental to dissect out the molecular mechanisms at the basis on neuronal cell death.
A wide range of cell models are available and can be produced for any specific disorders. These cell models may be very simple, such as fibroblasts, myoblasts, peripheral blood mononuclear cells (PBMC) (etc) derived from patients or any other type of primary culture cells (neurons, astrocytes, muscle, etc.) derived from animal models of the specific disease studies and maintained in culture, or artificially produced by inserting a mutant gene (or knocking down an endogenous gene) in selected cell type using transfection or infection procedures. The recently discover that fibroblasts (or PBMC) from patients can be reprogrammed to obtain pluripotent stem cells (iPSCs) which in turn can be differentiated to specific cell types has opened new way to study the neurodenegerative processes. In this lesson we will analyse and compare the different models available to conduct any type of biochemical, cellular and functional analysis in in vitro models of MN and NMJ diseases.
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