期刊
EXPERIMENTAL NEUROLOGY
卷 262, 期 -, 页码 138-151出版社
ACADEMIC PRESS INC ELSEVIER SCIENCE
DOI: 10.1016/j.expneurol.2014.07.001
关键词
Motor neuron disease; Motoneuron; Neurodegeneration; Therapy; Clinical trials
资金
- Fund for Scientific Research Flanders (FWD)
- Government agency for Innovation by Science and Technology (IWT)
- University of Leuven [GOA/11/014]
- Interuniversity Attraction Poles program of the Belgian Federal Science Policy Office [P7/16]
- European Community's Health Seventh Framework Programme
- Association Beige contre les Maladies neuro-Musculaires (ABMM)
- Association Francaise contre les Myopathies (AFM) [16525]
- Belgian ALS Liga
- ALS Therapy Alliance
- ALS Association (ALSA) [039CUK, 14-LGCA-181]
- Muscular Dystrophy Association (MDA) [MDA295317]
- E. von Behring Chair for Neuromuscular and Neurodegenerative Disorders
Amyotrophic lateral sclerosis (ALS) is characterized by the selective death of motor neurons in the motor cortex, brainstem and spinal cord. It is a neurodegenerative disorder with high genetic and phenotypic variability. In most patients, the cause of the disease is unknown. Until now, no treatment strategy has been discovered with the exception of riluzole which has a moderate effect on the disease process. While developing a new causal therapy targeting a specific disease-causing gene can have a huge effect on the disease process, only a limited number of ALS patients will benefit from such a therapy. Alternatively, pathogenic processes that are common in ALS patients with different etiology can also be targeted. The effect of such a modifying treatment will be smaller, but the target population will be larger as more ALS patients could benefit. In this review, we summarize the evidence for the involvement of different biological processes in the pathogenesis of ALS and will discuss how strategies influencing these processes can be translated into new therapeutic approaches. In order to further improve this translational step, there is an urgent need for a better understanding of the underlying mechanism(s), for new ALS animal models and for rigorous protocols to perform preclinical studies. (C) 2014 The Authors. Published by Elsevier Inc.
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