Stress granules in the spinal muscular atrophy and amyotrophic lateral sclerosis: The correlation and promising therapy

Neurobiology of Disease

Shanshan Mao¹  Jianhua Mao²  LiDan Hu³  Guannan Bai³  Li Lin³  Bingjie Liu³ 

[1] Corresponding authors.;State Key Laboratory of Membrane Biology, School of Life Sciences, Tsinghua University, Beijing 100084, China;the Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou 310052, China;

关键词: Stress granules;    Amyotrophic lateral sclerosis;    Spinal muscular atrophy;    RNA binding proteins;    Drug;   

DOI  :  

来源: DOAJ

【 摘 要 】

Increasing genetic and biochemical evidence has broadened our view of the pathomechanisms that lead to Spinal muscular atrophy (SMA) and Amyotrophic lateral sclerosis (ALS), two fatal neurodegenerative diseases with similar symptoms and causes. Stress granules are dynamic cytosolic storage hubs for mRNAs in response to stress exposures, that are evolutionarily conserved cytoplasmic RNA granules in somatic cells. A lot of previous studies have shown that the impaired stress granules are crucial events in SMA/ALS pathogenesis. In this review, we described the key stress granules related RNA binding proteins (SMN, TDP-43, and FUS) involved in SMA/ALS, summarized the reported mutations in these RNA binding proteins involved in SMA/ALS pathogenesis, and discussed the mechanisms through which stress granules dynamics participate in the diseases. Meanwhile, we described the applications and limitation of current therapies targeting SMA/ALS. We futher proposed the promising targets on stress granules in the future therapeutic interventions of SMA/ALS.

【 授权许可】

Unknown