TY - JOUR
T1 - The use of stem cells to model amyotrophic lateral sclerosis and frontotemporal dementia
T2 - from basic research to regenerative medicine
AU - Hedges, Erin Christine
AU - Mehler, Vera
AU - Nishimura, Agnes
PY - 2016/2
Y1 - 2016/2
N2 - In recent years several genes have linked amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) as a spectrum disease, however little is known about what triggers their onset. With the ability to generate patient specific stem cell lines from somatic cells, it is possible to model disease without the need to transfect cells with exogenous DNA. These pluripotent stem cells have opened new avenues for identification of disease phenotypes and their relation to specific molecular pathways. Thus, as never before, compounds with potential applications for regenerative medicine can be specifically tailored in patient derived cultures. In this review, we discuss how patient specific induced pluripotent stem cells (iPSCs) have been used to model ALS and FTD, and the most recent drug screening targets for these diseases. We also discuss how an iPSC bank would improve the quality of the available cell lines and how it would increase knowledge about the ALS/FTD disease spectrum.
AB - In recent years several genes have linked amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) as a spectrum disease, however little is known about what triggers their onset. With the ability to generate patient specific stem cell lines from somatic cells, it is possible to model disease without the need to transfect cells with exogenous DNA. These pluripotent stem cells have opened new avenues for identification of disease phenotypes and their relation to specific molecular pathways. Thus, as never before, compounds with potential applications for regenerative medicine can be specifically tailored in patient derived cultures. In this review, we discuss how patient specific induced pluripotent stem cells (iPSCs) have been used to model ALS and FTD, and the most recent drug screening targets for these diseases. We also discuss how an iPSC bank would improve the quality of the available cell lines and how it would increase knowledge about the ALS/FTD disease spectrum.
KW - stem cells
KW - Amyotrophic Lateral Sclerosis
KW - fronto temporal dementia
U2 - 10.1155/2016/9279516
DO - 10.1155/2016/9279516
M3 - Literature review
SN - 1687-9678
SP - 1
EP - 20
JO - Stem cells international
JF - Stem cells international
ER -