Pluripotent Stem Cells Models for Huntington's Disease: Prospects and Challenges

被引:10
作者
Carter, Richard L. [1 ,2 ,3 ]
Chan, Anthony W. S. [1 ,2 ,3 ]
机构
[1] Yerkes Natl Primate Res Ctr, Atlanta, GA 30329 USA
[2] Emory Univ, Grad Div Biol & Biomed Sci, Genet & Mol Biol Program, Atlanta, GA 30322 USA
[3] Emory Univ, Sch Med, Dept Human Genet, Atlanta, GA 30322 USA
关键词
Stem cell models; Huntington's disease; Induced pluripotent stem cells; Drug discovery; IN-VITRO; NEURODEGENERATIVE DISEASE; NEUROTROPHIC FACTOR; STEM/STROMAL CELLS; NEURAL TRANSPLANTS; DIFFERENTIATION; NEURONS; MECHANISMS; REPEAT; LINES;
D O I
10.1016/j.jgg.2012.04.006
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
Pluripotent cellular models have shown great promise in the study of a number of neurological disorders. Several advantages of using a stem cell model include the potential for cells to derive disease relevant neuronal cell types, providing a system for researchers to monitor disease progression during neurogenesis, along with serving as a platform for drug discovery. A number of stem cell derived models have been employed to establish in vitro research models of Huntington's disease that can be used to investigate cellular pathology and screen for drug and cell-based therapies. Although some progress has been made, there are a number of challenges and limitations that must be overcome before the true potential of this research strategy is achieved. In this article we review current stem cell models that have been reported, as well as discuss the issues that impair these studies. We also highlight the prospective application of Huntington's disease stem cell models in the development of novel therapeutic strategies and advancement of personalized medicine.
引用
收藏
页码:253 / 259
页数:7
相关论文
共 81 条
[1]   Effect of fetal neural transplants inpatients with Huntington's disease 6 years after surgery:: a long-term follow-up study [J].
Bachoud-Lévi, AC ;
Gaura, V ;
Brugières, P ;
Lefaucheur, JP ;
Boissé, MF ;
Maison, P ;
Baudic, S ;
Ribeiro, MJ ;
Bourdet, C ;
Remy, P ;
Cesaro, P ;
Hantraye, P ;
Peschanski, M .
LANCET NEUROLOGY, 2006, 5 (04) :303-309
[2]   Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice [J].
Boudreau, Ryan L. ;
McBride, Jodi L. ;
Martins, Ines ;
Shen, Shihao ;
Xing, Yi ;
Carter, Barrie J. ;
Davidson, Beverly L. .
MOLECULAR THERAPY, 2009, 17 (06) :1053-1063
[3]   Derivation of Huntington's Disease-Affected Human Embryonic Stem Cell Lines [J].
Bradley, Cara K. ;
Scott, Heather A. ;
Chami, Omar ;
Peura, Teija T. ;
Dumevska, Biljana ;
Schmidt, Uli ;
Stojanov, Tomas .
STEM CELLS AND DEVELOPMENT, 2011, 20 (03) :495-502
[4]   The first reported generation of several induced pluripotent stem cell lines from homozygous and heterozygous Huntington's disease patients demonstrates mutation related enhanced lysosomal activity [J].
Camnasio, Stefano ;
Carri, Alessia Delli ;
Lombardo, Angelo ;
Grad, Iwona ;
Mariotti, Caterina ;
Castucci, Alessia ;
Rozell, Bjorn ;
Lo Riso, Pietro ;
Castiglioni, Valentina ;
Zuccato, Chiara ;
Rochon, Christelle ;
Takashima, Yasuhiro ;
Diaferia, Giuseppe ;
Biunno, Ida ;
Gellera, Cinzia ;
Jaconi, Marisa ;
Smith, Austin ;
Hovatta, Outi ;
Naldini, Luigi ;
Di Donato, Stefano ;
Feki, Anis ;
Cattaneo, Elena .
NEUROBIOLOGY OF DISEASE, 2012, 46 (01) :41-51
[5]   Induced pluripotent stem cell lines from Huntington's disease mice undergo neuronal differentiation while showing alterations in the lysosomal pathway [J].
Castiglioni, Valentina ;
Onorati, Marco ;
Rochon, Christelle ;
Cattaneo, Elena .
NEUROBIOLOGY OF DISEASE, 2012, 46 (01) :30-40
[6]   Reprogramming Huntington Monkey Skin Cells into Pluripotent Stem Cells [J].
Chan, Anthony W. S. ;
Cheng, Pei-Hsun ;
Neumann, Adam ;
Yang, Jin-Jing .
CELLULAR REPROGRAMMING, 2010, 12 (05) :509-517
[7]   Transgenic Primate Research Paves the Path to a Better Animal Model: Are We a Step Closer to Curing Inherited Human Genetic Disorders? [J].
Chan, Anthony W. S. .
JOURNAL OF MOLECULAR CELL BIOLOGY, 2009, 1 (01) :13-14
[8]   Neural transplants in patients with Huntington's disease undergo disease-like neuronal degeneration [J].
Cicchetti, F. ;
Saporta, S. ;
Hauser, R. A. ;
Parent, M. ;
Saint-Pierre, M. ;
Sanberg, P. R. ;
Li, X. J. ;
Parker, J. R. ;
Chu, Y. ;
Mufson, E. J. ;
Kordower, J. H. ;
Freeman, T. B. .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2009, 106 (30) :12483-12488
[9]   Neural stem cell systems: physiological players or in vitro entities? [J].
Conti, Luciano ;
Cattaneo, Elena .
NATURE REVIEWS NEUROSCIENCE, 2010, 11 (03) :176-187
[10]   Huntington's Disease: Can Mice Lead the Way to Treatment? [J].
Crook, Zachary R. ;
Housman, David .
NEURON, 2011, 69 (03) :423-435