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báo cáo khoa học: " Capturing Alzheimer’s disease genomes with induced pluripotent stem cells: prospects and challenges"

Tuyển tập báo cáo các nghiên cứu khoa học quốc tế ngành y học dành cho các bạn tham khảo đề tài: Capturing Alzheimer’s disease genomes with induced pluripotent stem cells: prospects and challenges | Israel and Goldstein Genome Medicine 2011 3 49 http genomemedicine.eom content 3 7 49 w Genome Medicine REVIEW L_ Capturing Alzheimer s disease genomes with induced pluripotent stem cells prospects and challenges Mason A Israel and Lawrence SB Goldstein Abstract A crucial limitation to our understanding of Alzheimer s disease AD is the inability to test hypotheses on live patient-specific neurons. Patient autopsies are limited in supply and only reveal endpoints of disease. Rodent models harboring familial AD mutations lack important pathologies and animal models have not been useful in modeling the sporadic form of AD because of complex genetics. The recent development of induced pluripotent stem cells iPSCs provides a method to create live patient-specific models of disease and to investigate disease phenotypes in vitro. In this review we discuss the genetics of AD patients and the potential for iPSCs to capture the genomes of these individuals and generate relevant cell types. Specifically we examine recent insights into the genetic fidelity of iPSCs advances in the area of neuronal differentiation and the ability of iPSCs to model neurodegenerative diseases. Introduction from AD patient genome to disease in a dish Alzheimer s disease AD is a common fatal neurode-generative disease that currently afflicts more than 35 million people worldwide 1 . With the increasing longevity and aging of many populations around the world the devastation caused by AD to patients their families societies and economies is growing. Currently there is no approved treatment with a proven diseasemodifying effect 2 . Mechanistic studies of AD generally rely on autopsy samples which are limited in supply and contain the disease aftermath or on animal models which do not Correspondence misrael@ucsd.edu lgoldstein@ucsd.edu Department of Cellular and Molecular Medicine University of California San Diego La Jolla CA 92093 USA 2 BioMed Central 2011 BioMed Central Ltd fully recapitulate