4.6 Review

A perspective on therapies for amyotrophic lateral sclerosis: can disease progression be curbed?

期刊

TRANSLATIONAL NEURODEGENERATION
卷 10, 期 1, 页码 -

出版社

BMC
DOI: 10.1186/s40035-021-00250-5

关键词

Amyotrophic lateral sclerosis; Motor neurons; Autophagy; Stem cells; Gene editing

资金

  1. National Nature Research Foundation [81671241, 81771521]
  2. Shanghai Shuguang Plan Project [18SG15]
  3. Shanghai outstanding young scholars Project

向作者/读者索取更多资源

ALS is a progressive neurodegenerative disease with limited treatment options currently available. While riluzole has shown modest efficacy, there is still debate on the effectiveness of edaravone in slowing disease progression. Impaired autophagy plays a critical role in ALS pathogenesis, highlighting the need for novel therapeutic strategies.
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease involving both upper and lower motor neurons, leading to paralysis and eventually death. Symptomatic treatments such as inhibition of salivation, alleviation of muscle cramps, and relief of spasticity and pain still play an important role in enhancing the quality of life. To date, riluzole and edaravone are the only two drugs approved by the Food and Drug Administration for the treatment of ALS in a few countries. While there is adequate consensus on the modest efficacy of riluzole, there are still open questions concerning the efficacy of edaravone in slowing the disease progression. Therefore, identification of novel therapeutic strategies is urgently needed. Impaired autophagic process plays a critical role in ALS pathogenesis. In this review, we focus on therapies modulating autophagy in the context of ALS. Furthermore, stem cell therapies, gene therapies, and newly-developed biomaterials have great potentials in alleviating neurodegeneration, which might halt the disease progression. In this review, we will summarize the current and prospective therapies for ALS.

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