Application of antisense oligonucleotide drugs in amyotrophic lateral sclerosis and Huntington’s disease
Abstract Amyotrophic lateral sclerosis (ALS) and Huntington’s disease (HD) are diverse in clinical presentation and are caused by complex and multiple factors, including genetic mutations and environmental factors. Numerous therapeutic approaches have been developed based on the genetic causes and p...
Saved in:
| Main Authors: | , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
BMC
2025-01-01
|
| Series: | Translational Neurodegeneration |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s40035-025-00466-9 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1832585434968883200 |
|---|---|
| author | Kaili Ou Qingqing Jia Dandan Li Shihua Li Xiao-Jiang Li Peng Yin |
| author_facet | Kaili Ou Qingqing Jia Dandan Li Shihua Li Xiao-Jiang Li Peng Yin |
| author_sort | Kaili Ou |
| collection | DOAJ |
| description | Abstract Amyotrophic lateral sclerosis (ALS) and Huntington’s disease (HD) are diverse in clinical presentation and are caused by complex and multiple factors, including genetic mutations and environmental factors. Numerous therapeutic approaches have been developed based on the genetic causes and potential mechanisms of ALS and HD. Currently, available treatments for various neurodegenerative diseases can alleviate symptoms but do not provide a definitive cure. Gene therapy, which aims to modify or express specific proteins for neuroprotection or correction, is considered a powerful tool in managing neurodegenerative conditions. To date, antisense oligonucleotide (ASO) drugs targeting the pathological genes associated with ALS and HD have shown promising results in numerous animal studies and several clinical trials. This review provides a comprehensive overview of the development, mechanisms of action, limitations, and clinical applications of ASO drugs in neurodegenerative diseases, with a specific focus on ALS and HD therapeutic strategies. |
| format | Article |
| id | doaj-art-83e36cdfdd904c5e90721eb2a8a56312 |
| institution | Kabale University |
| issn | 2047-9158 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | BMC |
| record_format | Article |
| series | Translational Neurodegeneration |
| spelling | doaj-art-83e36cdfdd904c5e90721eb2a8a563122025-01-26T12:49:56ZengBMCTranslational Neurodegeneration2047-91582025-01-0114111310.1186/s40035-025-00466-9Application of antisense oligonucleotide drugs in amyotrophic lateral sclerosis and Huntington’s diseaseKaili Ou0Qingqing Jia1Dandan Li2Shihua Li3Xiao-Jiang Li4Peng Yin5State Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Key Laboratory of Non-Human Primate Research, Key Laboratory of CNS Regeneration (Ministry of Education), Guangdong-Hongkong-Macau Institute of CNS Regeneration, Jinan UniversityState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Key Laboratory of Non-Human Primate Research, Key Laboratory of CNS Regeneration (Ministry of Education), Guangdong-Hongkong-Macau Institute of CNS Regeneration, Jinan UniversityState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Key Laboratory of Non-Human Primate Research, Key Laboratory of CNS Regeneration (Ministry of Education), Guangdong-Hongkong-Macau Institute of CNS Regeneration, Jinan UniversityState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Key Laboratory of Non-Human Primate Research, Key Laboratory of CNS Regeneration (Ministry of Education), Guangdong-Hongkong-Macau Institute of CNS Regeneration, Jinan UniversityState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Key Laboratory of Non-Human Primate Research, Key Laboratory of CNS Regeneration (Ministry of Education), Guangdong-Hongkong-Macau Institute of CNS Regeneration, Jinan UniversityState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Key Laboratory of Non-Human Primate Research, Key Laboratory of CNS Regeneration (Ministry of Education), Guangdong-Hongkong-Macau Institute of CNS Regeneration, Jinan UniversityAbstract Amyotrophic lateral sclerosis (ALS) and Huntington’s disease (HD) are diverse in clinical presentation and are caused by complex and multiple factors, including genetic mutations and environmental factors. Numerous therapeutic approaches have been developed based on the genetic causes and potential mechanisms of ALS and HD. Currently, available treatments for various neurodegenerative diseases can alleviate symptoms but do not provide a definitive cure. Gene therapy, which aims to modify or express specific proteins for neuroprotection or correction, is considered a powerful tool in managing neurodegenerative conditions. To date, antisense oligonucleotide (ASO) drugs targeting the pathological genes associated with ALS and HD have shown promising results in numerous animal studies and several clinical trials. This review provides a comprehensive overview of the development, mechanisms of action, limitations, and clinical applications of ASO drugs in neurodegenerative diseases, with a specific focus on ALS and HD therapeutic strategies.https://doi.org/10.1186/s40035-025-00466-9Amyotrophic lateral sclerosisHuntington’s diseaseClinical trialAntisense oligonucleotide |
| spellingShingle | Kaili Ou Qingqing Jia Dandan Li Shihua Li Xiao-Jiang Li Peng Yin Application of antisense oligonucleotide drugs in amyotrophic lateral sclerosis and Huntington’s disease Translational Neurodegeneration Amyotrophic lateral sclerosis Huntington’s disease Clinical trial Antisense oligonucleotide |
| title | Application of antisense oligonucleotide drugs in amyotrophic lateral sclerosis and Huntington’s disease |
| title_full | Application of antisense oligonucleotide drugs in amyotrophic lateral sclerosis and Huntington’s disease |
| title_fullStr | Application of antisense oligonucleotide drugs in amyotrophic lateral sclerosis and Huntington’s disease |
| title_full_unstemmed | Application of antisense oligonucleotide drugs in amyotrophic lateral sclerosis and Huntington’s disease |
| title_short | Application of antisense oligonucleotide drugs in amyotrophic lateral sclerosis and Huntington’s disease |
| title_sort | application of antisense oligonucleotide drugs in amyotrophic lateral sclerosis and huntington s disease |
| topic | Amyotrophic lateral sclerosis Huntington’s disease Clinical trial Antisense oligonucleotide |
| url | https://doi.org/10.1186/s40035-025-00466-9 |
| work_keys_str_mv | AT kailiou applicationofantisenseoligonucleotidedrugsinamyotrophiclateralsclerosisandhuntingtonsdisease AT qingqingjia applicationofantisenseoligonucleotidedrugsinamyotrophiclateralsclerosisandhuntingtonsdisease AT dandanli applicationofantisenseoligonucleotidedrugsinamyotrophiclateralsclerosisandhuntingtonsdisease AT shihuali applicationofantisenseoligonucleotidedrugsinamyotrophiclateralsclerosisandhuntingtonsdisease AT xiaojiangli applicationofantisenseoligonucleotidedrugsinamyotrophiclateralsclerosisandhuntingtonsdisease AT pengyin applicationofantisenseoligonucleotidedrugsinamyotrophiclateralsclerosisandhuntingtonsdisease |