System Maintenance Notice

Please note that maintenance work is scheduled between 4 AM and 5 AM - CET. During this time, you may experience reduced performance. Thanks for your patience.

Publications

Journal Article Review Epublish

PubMed: 40234983 DOI: 10.1186/s40035-025-00477-6

Gene therapy breakthroughs in ALS: a beacon of hope for 20% of ALS patients.

Transl Neurodegener 14(1):19

Authors: Qingjian Xie, Kezheng Li, Yinuo Chen, Yaojia Li, Wenhua Jiang, Wen Cao, Huan Yu, Dongsheng Fan, Binbin Deng

Affiliations

  • Department of Neurology, First Affiliated Hospital of Wenzhou Medical University, Wenzhou, 32500, China. First School of Clinical Medicine, Wenzhou Medical University, Wenzhou, China. Zhejiang Key Laboratory of Intelligent Cancer Biomarker Discovery and Translation, First Affiliated Hospital of Wenzhou Medical University, Wenzhou, China.
  • Department of Neurology, First Affiliated Hospital of Wenzhou Medical University, Wenzhou, 32500, China. First School of Clinical Medicine, Wenzhou Medical University, Wenzhou, China.
  • Department of Neurology, First Affiliated Hospital of Wenzhou Medical University, Wenzhou, 32500, China.
  • Department of Neurology, Peking University Third Hospital, Beijing, China.
  • Department of Pediatrics, Second Affiliated Hospital and Yuying Children'S Hospital of Wenzhou Medical University, Wenzhou, China.
  • Department of Rehabilitation, The First Affiliated Hospital of Wenzhou Medical University, Wenzhou, China. Department of Neurology, First Affiliated Hospital of Wenzhou Medical University, Wenzhou, 32500, China.

Abstract

Amyotrophic lateral sclerosis (ALS) is a fatal motor neuron disease that remains incurable. Although the etiologies of ALS are diverse and the precise pathogenic mechanisms are not fully understood, approximately 20% of ALS cases are caused by genetic factors. Therefore, advancing targeted gene therapies holds significant promise, at least for the 20% of ALS patients with genetic etiologies. In this review, we summarize the main strategies and techniques of current ALS gene therapies based on ALS risk genes, and review recent findings from animal studies and clinical trials. Additionally, we highlight ALS-related genes with well-understood pathogenic mechanisms and the potential of numerous emerging gene-targeted therapeutic approaches for ALS.

MeSH

Genes, Therapeutics

Cited by