Stem cell contributions to neurological disease modeling and personalized medicine
Nicholas Liang1, Cleber A Trujillo1, Priscilla D Negraes1
1University of California San Diego, School of Medicine, Department of Pediatrics/Rady Children's Hospital San Diego, Stem Cell Program, La Jolla, CA 92093, USA.
Abstract:
Human induced pluripotent stem cells (iPSCs) represent a revolutionary tool for disease modeling and drug discovery. The generation of tissue-relevant cell types exhibiting a patient's genetic and molecular background offers the ability to develop individual and effective therapies. In this review, we present some major achievements in the neuroscience field using iPSCs and discuss promising perspectives in personalized medicine. In addition to disease modeling, the understanding of the cellular and molecular basis of neurological disorders is explored, including the discovery of new targets and potential drugs. Ultimately, we highlight how iPSC technology, together with genome editing approaches, may bring a deep impact on pre-clinical trials by reducing costs and increasing the success of treatments in a personalized fashion.
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