Progress in gene therapy mechanism of amyotrophic lateral sclerosis
Gene therapy research on amyotrophic lateral sclerosis (ALS) focuses on SOD1, C9ORF72, FUS and ATAX2, et al. According to the mechanism, the pathogenicity of variant genes is mostly acquired by the toxicity gain of function. Two strategies are adopted for the silencing and editing of disease causing...
| Main Author: | |
|---|---|
| Format: | Article |
| Language: | zho |
| Published: |
Institute of Basic Medical Sciences and Peking Union Medical College Hospital, Chinese Academy of Medical Sciences / Peking Union Medical College.
2023-04-01
|
| Series: | Jichu yixue yu linchuang |
| Subjects: | |
| Online Access: | http://journal11.magtechjournal.com/Jwk_jcyxylc/fileup/1001-6325/PDF/1001-6325-2023-43-4-674.pdf |
| _version_ | 1827391071156436992 |
|---|---|
| author | LI Xiaoguang, YANG Lu, LIU Xudong, JIA Xinmiao, YANG Xinzhuang, CUI Liying |
| author_facet | LI Xiaoguang, YANG Lu, LIU Xudong, JIA Xinmiao, YANG Xinzhuang, CUI Liying |
| author_sort | LI Xiaoguang, YANG Lu, LIU Xudong, JIA Xinmiao, YANG Xinzhuang, CUI Liying |
| collection | DOAJ |
| description | Gene therapy research on amyotrophic lateral sclerosis (ALS) focuses on SOD1, C9ORF72, FUS and ATAX2, et al. According to the mechanism, the pathogenicity of variant genes is mostly acquired by the toxicity gain of function. Two strategies are adopted for the silencing and editing of disease causing genes, namely, naked injection of antisense oligonucleotides (ASO) to induce the degradation of mRNA mediated by RNase H or the silencing of disease causing gene mediated by adeno-associated virus (AAV) to delivery RNA interference (RNAi) to decrease toxicity protein. CRISPR/Cas9 edits and modifies the causing gene to decrease the expression of toxic protein. In addition, there has been much progress in decreasing the level of toxic protein through specific antibodies and metabolic regulators, as well as neuroprotective therapy. |
| first_indexed | 2024-03-08T17:03:23Z |
| format | Article |
| id | doaj.art-cd8b5dd674ab421ab5afd24ba7219eb6 |
| institution | Directory Open Access Journal |
| issn | 1001-6325 |
| language | zho |
| last_indexed | 2024-03-08T17:03:23Z |
| publishDate | 2023-04-01 |
| publisher | Institute of Basic Medical Sciences and Peking Union Medical College Hospital, Chinese Academy of Medical Sciences / Peking Union Medical College. |
| record_format | Article |
| series | Jichu yixue yu linchuang |
| spelling | doaj.art-cd8b5dd674ab421ab5afd24ba7219eb62024-01-04T07:32:42ZzhoInstitute of Basic Medical Sciences and Peking Union Medical College Hospital, Chinese Academy of Medical Sciences / Peking Union Medical College.Jichu yixue yu linchuang1001-63252023-04-0143467467910.16352/j.issn.1001-6325.2023.04.0674Progress in gene therapy mechanism of amyotrophic lateral sclerosisLI Xiaoguang, YANG Lu, LIU Xudong, JIA Xinmiao, YANG Xinzhuang, CUI Liying01. Department of Neurology;;2. Centre for Medical Science Research,Peking Union Medical College Hospital, CAMS & PUMC, Beijing 100730,ChinaGene therapy research on amyotrophic lateral sclerosis (ALS) focuses on SOD1, C9ORF72, FUS and ATAX2, et al. According to the mechanism, the pathogenicity of variant genes is mostly acquired by the toxicity gain of function. Two strategies are adopted for the silencing and editing of disease causing genes, namely, naked injection of antisense oligonucleotides (ASO) to induce the degradation of mRNA mediated by RNase H or the silencing of disease causing gene mediated by adeno-associated virus (AAV) to delivery RNA interference (RNAi) to decrease toxicity protein. CRISPR/Cas9 edits and modifies the causing gene to decrease the expression of toxic protein. In addition, there has been much progress in decreasing the level of toxic protein through specific antibodies and metabolic regulators, as well as neuroprotective therapy.http://journal11.magtechjournal.com/Jwk_jcyxylc/fileup/1001-6325/PDF/1001-6325-2023-43-4-674.pdfamyotrophic lateral sclerosis|gene therapy|antisense oligonucleotides|rna interference|crispr-cas9 |
| spellingShingle | LI Xiaoguang, YANG Lu, LIU Xudong, JIA Xinmiao, YANG Xinzhuang, CUI Liying Progress in gene therapy mechanism of amyotrophic lateral sclerosis Jichu yixue yu linchuang amyotrophic lateral sclerosis|gene therapy|antisense oligonucleotides|rna interference|crispr-cas9 |
| title | Progress in gene therapy mechanism of amyotrophic lateral sclerosis |
| title_full | Progress in gene therapy mechanism of amyotrophic lateral sclerosis |
| title_fullStr | Progress in gene therapy mechanism of amyotrophic lateral sclerosis |
| title_full_unstemmed | Progress in gene therapy mechanism of amyotrophic lateral sclerosis |
| title_short | Progress in gene therapy mechanism of amyotrophic lateral sclerosis |
| title_sort | progress in gene therapy mechanism of amyotrophic lateral sclerosis |
| topic | amyotrophic lateral sclerosis|gene therapy|antisense oligonucleotides|rna interference|crispr-cas9 |
| url | http://journal11.magtechjournal.com/Jwk_jcyxylc/fileup/1001-6325/PDF/1001-6325-2023-43-4-674.pdf |
| work_keys_str_mv | AT lixiaoguangyangluliuxudongjiaxinmiaoyangxinzhuangcuiliying progressingenetherapymechanismofamyotrophiclateralsclerosis |