Genetically-Modified Human Pluripotent Stem Cells: New Hopes for the Understanding and the Treatment of Neurological Diseases?
The fundamental inaccessibility of the human neural cell types affected by neurological disorders prevents their
isolation for in vitro studies of disease mechanisms or for drug screening efforts. Pluripotent stem cells represent a new
interesting way to generate models of human neurological disorders, explore the physiopathological mechanisms and develop
new therapeutic strategies. Disease-specific human embryonic stem cells were the first source of material to be used
to study certain disease states. The recent demonstration that human somatic cells, such as fibroblasts or blood cells, can
be genetically converted to induced pluripotent stem cells (hiPSCs) together with the continuous improvement of methods
to differentiate these cells into disease-affected neuronal subtypes opens new perspectives to model and understand a large
number of human pathologies. This review focuses on the opportunities concerning the use disease-specific human pluripotent
stem cells as well as the different challenges that still need to be overcome. We also discuss the recent improvements
in the genetic manipulation of human pluripotent stem cells and the consequences of these on disease modeling and
drug screening for neurological diseases.
Keywords: Human induced pluripotent stem cells, human embryonic stem cells, genetic diseases, disease modeling, drug
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