Induced pluripotent stem cells as a model for accelerated patient-and disease-specific drug discovery

I Gunaseeli, MX Doss, C Antzelevitch… - Current medicinal …, 2010 - ingentaconnect.com
I Gunaseeli, MX Doss, C Antzelevitch, J Hescheler, A Sachinidis
Current medicinal chemistry, 2010ingentaconnect.com
Human induced pluripotent stem (iPS) cells hold great promise for therapy of a number of
degenerative diseases such as ischemic heart failure, Parkinson's disease, Alzheimer's
disease, diabetes mellitus, sickle cell anemia and Huntington disease. They also have the
potential to accelerate drug discovery in 3 ways. The first involves the delineation of
chemical components for efficient reprogramming of patient's blood cells or cells from
biopsies, obviating the need for cellular delivery of reprogramming exogenous transgenes …
Human induced pluripotent stem (iPS) cells hold great promise for therapy of a number of degenerative diseases such as ischemic heart failure, Parkinson's disease, Alzheimer's disease, diabetes mellitus, sickle cell anemia and Huntington disease. They also have the potential to accelerate drug discovery in 3 ways. The first involves the delineation of chemical components for efficient reprogramming of patient's blood cells or cells from biopsies, obviating the need for cellular delivery of reprogramming exogenous transgenes, thereby converting hope into reality for patients suffering from degenerative diseases. Patients worldwide stand to benefit from the clinical applicability of iPS cell-based cell replacement therapy for a number of degenerative diseases. The second is the potential for discovering novel drugs in a high throughput manner using patient-specific iPS cell-derived somatic cells possessing the etiology of the specific disease. The third is their suitability for toxicological testing of drugs and environmental factors. This review focuses on these potential applications of iPS cells with special emphasis on recent updates of iPS cell research contributing to the accelerated drug discovery.
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