Gene Silencing and Editing Strategies for Neurodegenerative Diseases
Neurodegenerative diseases (NDs) are a heterogeneous group of disorders affecting the central nervous system. Despite significant differences in their causes, neuropathological abnormalities, and clinical outcomes, some similarities can be found among them, as for example: 1) frequent aggregation an...
I tiakina i:
| Ngā kaituhi matua: | , |
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| Hōputu: | Online |
| Reo: | Ingarihi |
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Frontiers Media SA
2021
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| Ngā marau: | |
| Urunga tuihono: | 31961 |
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Kāore He Tūtohu, Me noho koe te mea tuatahi ki te tūtohu i tēnei pūkete!
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| _version_ | 1869524142817017856 |
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| author | Clévio Nóbrega Sandro Alves |
| author_browse | Clévio Nóbrega Sandro Alves |
| author_facet | Clévio Nóbrega Sandro Alves |
| author_sort | Clévio Nóbrega |
| collection | Directory of Open Access Books |
| description | Neurodegenerative diseases (NDs) are a heterogeneous group of disorders affecting the central nervous system. Despite significant differences in their causes, neuropathological abnormalities, and clinical outcomes, some similarities can be found among them, as for example: 1) frequent aggregation and deposition of misfolded proteins, 2) common molecular mechanisms leading to neurodegeneration, and 3) certain overlap in symptoms and clinical features. To date, there is no cure that could stop or delay the progression of these diseases. The advent of advanced gene therapy techniques such as gene silencing and gene editing opened a new avenue for the development of therapeutic strategies for NDs. The discovery of the RNA interference (RNAi) mechanism, in 1998, by Andrew Fire and Craig Mello allowed an important boost to the gene therapy field, providing a potential therapeutic strategy to treat inherited dominant genetic disorders. The use of small RNA sequences to control the expression of disease-causing genes rapidly implemented in the preclinical studies for different diseases. In the field of NDs, several successful studies using this technology proved its potential as a therapeutic option. However, issues like the type of delivery system (non-viral versus viral) or the potential toxicity of the small RNA molecules, made the translation of gene silencing therapeutics to human application very slow and difficult. Recently, a new hope in the gene therapy field emerged with the development of gene editing techniques like TALENs or CRISPR/Cas9 systems. The opportunity of editing or deleting gene sequences drove the scientific community euphoric, with an enormous increase in the number of published studies using this type of techniques. Recently, the first clinical trial using one of these systems was approved in China. For NDs, gene-editing technology also represents an important therapeutic option, and the first preclinical studies are now being published, showing the potential accomplishment for this technology. |
| format | Online |
| id | doab-20.500.12854ir-48351 |
| institution | Directory of Open Access Books |
| language | eng |
| publishDate | 2021 |
| publishDateRange | 2021 |
| publishDateSort | 2021 |
| publisher | Frontiers Media SA |
| publisherStr | Frontiers Media SA |
| record_format | ojs |
| spelling | doab-20.500.12854ir-483512024-04-05T17:30:43Z Gene Silencing and Editing Strategies for Neurodegenerative Diseases Clévio Nóbrega Sandro Alves RC321-571 Q1-390 Gene silencing Long non-coding RNAs RNA interference Neurodegenerative diseases CRISPR/Cas9 Neurodegeneration Gene editing Antisense oligonucleotides Neuroinflammation iPS cells thema EDItEUR::P Mathematics and Science::PS Biology, life sciences::PSA Life sciences: general issues::PSAN Neurosciences Neurodegenerative diseases (NDs) are a heterogeneous group of disorders affecting the central nervous system. Despite significant differences in their causes, neuropathological abnormalities, and clinical outcomes, some similarities can be found among them, as for example: 1) frequent aggregation and deposition of misfolded proteins, 2) common molecular mechanisms leading to neurodegeneration, and 3) certain overlap in symptoms and clinical features. To date, there is no cure that could stop or delay the progression of these diseases. The advent of advanced gene therapy techniques such as gene silencing and gene editing opened a new avenue for the development of therapeutic strategies for NDs. The discovery of the RNA interference (RNAi) mechanism, in 1998, by Andrew Fire and Craig Mello allowed an important boost to the gene therapy field, providing a potential therapeutic strategy to treat inherited dominant genetic disorders. The use of small RNA sequences to control the expression of disease-causing genes rapidly implemented in the preclinical studies for different diseases. In the field of NDs, several successful studies using this technology proved its potential as a therapeutic option. However, issues like the type of delivery system (non-viral versus viral) or the potential toxicity of the small RNA molecules, made the translation of gene silencing therapeutics to human application very slow and difficult. Recently, a new hope in the gene therapy field emerged with the development of gene editing techniques like TALENs or CRISPR/Cas9 systems. The opportunity of editing or deleting gene sequences drove the scientific community euphoric, with an enormous increase in the number of published studies using this type of techniques. Recently, the first clinical trial using one of these systems was approved in China. For NDs, gene-editing technology also represents an important therapeutic option, and the first preclinical studies are now being published, showing the potential accomplishment for this technology. 2021-02-11T14:20:37Z 2021-02-11T14:20:37Z 2019-01-23 14:53:42 2018 book 31961 16648714 9782889455515 https://directory.doabooks.org/handle/20.500.12854/48351 eng Frontiers Research Topics image/jpeg Attribution 4.0 International https://www.frontiersin.org/research-topics/5818/gene-silencing-and-editing-strategies-for-neurodegenerative-diseases Frontiers Media SA 10.3389/978-2-88945-551-5 10.3389/978-2-88945-551-5 bf5ce210-e72e-4860-ba9b-c305640ff3ae 9782889455515 115 open access |
| spellingShingle | RC321-571 Q1-390 Gene silencing Long non-coding RNAs RNA interference Neurodegenerative diseases CRISPR/Cas9 Neurodegeneration Gene editing Antisense oligonucleotides Neuroinflammation iPS cells thema EDItEUR::P Mathematics and Science::PS Biology, life sciences::PSA Life sciences: general issues::PSAN Neurosciences Clévio Nóbrega Sandro Alves Gene Silencing and Editing Strategies for Neurodegenerative Diseases |
| title | Gene Silencing and Editing Strategies for Neurodegenerative Diseases |
| title_full | Gene Silencing and Editing Strategies for Neurodegenerative Diseases |
| title_fullStr | Gene Silencing and Editing Strategies for Neurodegenerative Diseases |
| title_full_unstemmed | Gene Silencing and Editing Strategies for Neurodegenerative Diseases |
| title_short | Gene Silencing and Editing Strategies for Neurodegenerative Diseases |
| title_sort | gene silencing and editing strategies for neurodegenerative diseases |
| topic | RC321-571 Q1-390 Gene silencing Long non-coding RNAs RNA interference Neurodegenerative diseases CRISPR/Cas9 Neurodegeneration Gene editing Antisense oligonucleotides Neuroinflammation iPS cells thema EDItEUR::P Mathematics and Science::PS Biology, life sciences::PSA Life sciences: general issues::PSAN Neurosciences |
| topic_facet | RC321-571 Q1-390 Gene silencing Long non-coding RNAs RNA interference Neurodegenerative diseases CRISPR/Cas9 Neurodegeneration Gene editing Antisense oligonucleotides Neuroinflammation iPS cells thema EDItEUR::P Mathematics and Science::PS Biology, life sciences::PSA Life sciences: general issues::PSAN Neurosciences |
| url | 31961 |
| work_keys_str_mv | AT clevionobrega genesilencingandeditingstrategiesforneurodegenerativediseases AT sandroalves genesilencingandeditingstrategiesforneurodegenerativediseases |