Neurodegenerative disease: models, mechanisms, and a new hope

被引:507
作者
Gitler, Aaron D. [1 ]
Dhillon, Paraminder [2 ]
Shorter, James [3 ]
机构
[1] Stanford Univ, Sch Med, Dept Genet, Stanford, CA 94404 USA
[2] Co Biol, Dis Models & Mech, Cambridge CB24 9LF, England
[3] Univ Penn, Perelman Sch Med, Dept Biochem & Biophys, Philadelphia, PA USA
关键词
SPINAL MUSCULAR-ATROPHY; ALPHA-SYNUCLEIN; STEM-CELLS; LIFE-SPAN; ALS; TOXICITY; NEURONS; YEAST; AGGREGATION; ASTROCYTES;
D O I
10.1242/dmm.030205
中图分类号
Q2 [细胞生物学];
学科分类号
071009 ; 090102 ;
摘要
Neurodegeneration is a feature of many debilitating, incurable diseases that are rapidly rising in prevalence, such as Parkinson's disease. There is an urgent need to develop new and more effective therapeutic strategies to combat these devastating diseases. Models - from cell-based systems, to unicellular organisms, to complex animals - have proven to be a useful tool to help the research community shed light on the mechanisms underlying neurodegenerative diseases, and these advances have now begun to provide promising therapeutic avenues. In this themed issue of Disease Models & Mechanisms, a special collection of articles focused on neurodegenerative diseases is introduced. The collection includes original research articles that provide new insights into the complex pathophysiology of such diseases, revealing candidate biomarkers or therapeutic targets. Some of the articles describe a new disease model that enables deeper exploration of key mechanisms. We also present a series of reviews that highlight some of the recent translational advances made in studies of neurodegenerative diseases. In this Editorial, we summarize the articles featured in this collection, emphasizing the impact that model-based studies have made in this exciting area of research.
引用
收藏
页码:499 / 502
页数:4
相关论文
共 52 条
[21]   Constructing and Deconstructing Stem Cell Models of Neurological Disease [J].
Han, Steve S. W. ;
Williams, Luis A. ;
Eggan, Kevin C. .
NEURON, 2011, 70 (04) :626-644
[22]   Susan Lee Lindquist (1949-2016)-pioneer in the study of cellular protein folding and disease Obituary [J].
Hartl, Franz-Ulrich .
EMBO JOURNAL, 2016, 35 (24) :2626-2627
[23]   NEURODEGENERATIVE DISEASES [J].
Heemels, Marie-Therese .
NATURE, 2016, 539 (7628) :179-179
[24]   Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model [J].
Hua, Yimin ;
Sahashi, Kentaro ;
Rigo, Frank ;
Hung, Gene ;
Horev, Guy ;
Bennett, C. Frank ;
Krainer, Adrian R. .
NATURE, 2011, 478 (7367) :123-126
[25]   Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model [J].
Hua, Yimin ;
Sahashi, Kentaro ;
Hung, Gene ;
Rigo, Frank ;
Passini, Marco A. ;
Bennett, C. Frank ;
Krainer, Adrian R. .
GENES & DEVELOPMENT, 2010, 24 (15) :1634-1644
[26]   Non-cell autonomous toxicity in neurodegenerative disorders: ALS and beyond [J].
Ilieva, Hristelina ;
Polymenidou, Magdalini ;
Cleveland, Don W. .
JOURNAL OF CELL BIOLOGY, 2009, 187 (06) :761-772
[27]   Modifiers of C9orf72 dipeptide repeat toxicity connect nucleocytoplasmic transport defects to FTD/ALS [J].
Jovicic, Ana ;
Mertens, Jerome ;
Boeynaems, Steven ;
Bogaert, Elke ;
Chai, Noori ;
Yamada, Shizuka B. ;
Paul, Joseph W., III ;
Sun, Shuying ;
Herdy, Joseph R. ;
Bieri, Gregor ;
Kramer, Nicholas J. ;
Gage, Fred H. ;
Van den Bosch, Ludo ;
Robberecht, Wim ;
Gitler, Aaron D. .
NATURE NEUROSCIENCE, 2015, 18 (09) :1226-+
[28]   From Yeast to Patients: The Audacity and Vision of Susan Lindquist [J].
Khurana, Vikram ;
Chung, Chee Yeun ;
Tardiff, Daniel F. .
CELL SYSTEMS, 2017, 4 (02) :147-148
[29]   The tyrosine kinase receptor Tyro3 enhances lifespan and neuropeptide Y (Npy) neuron survival in the mouse anorexia (anx) mutation [J].
Kim, Dennis Y. ;
Yu, Joanna ;
Mui, Ryan K. ;
Niibori, Rieko ;
Bin Taufique, Hamza ;
Aslam, Rukhsana ;
Semple, John W. ;
Cordes, Sabine P. .
DISEASE MODELS & MECHANISMS, 2017, 10 (05) :581-595
[30]   Aggregation of huntingtin in yeast varies with the length of the polyglutamine expansion and the expression of chaperone proteins [J].
Krobitsch, S ;
Lindquist, S .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2000, 97 (04) :1589-1594