Nucleic acid-based therapeutics for the treatment of central nervous system disorders
Nucleic acid-based therapeutics (NBTs) are an emerging class of drugs with potential for the treatment of a wide range of central nervous system conditions. To date, pertaining to CNS indications, there are two commercially available NBTs and a large number of ongoing clinical trials. However, these...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2023-08-01
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| Series: | Frontiers in Genetics |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fgene.2023.1250276/full |
| _version_ | 1797741681651482624 |
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| author | Robyn McCartan Robyn McCartan Olga Khorkova Olga Khorkova Claude-Henry Volmar Claude-Henry Volmar Claes Wahlestedt Claes Wahlestedt |
| author_facet | Robyn McCartan Robyn McCartan Olga Khorkova Olga Khorkova Claude-Henry Volmar Claude-Henry Volmar Claes Wahlestedt Claes Wahlestedt |
| author_sort | Robyn McCartan |
| collection | DOAJ |
| description | Nucleic acid-based therapeutics (NBTs) are an emerging class of drugs with potential for the treatment of a wide range of central nervous system conditions. To date, pertaining to CNS indications, there are two commercially available NBTs and a large number of ongoing clinical trials. However, these NBTs are applied directly to the brain due to very low blood brain barrier permeability. In this review, we outline recent advances in chemical modifications of NBTs and NBT delivery techniques intended to promote brain exposure, efficacy, and possible future systemic application. |
| first_indexed | 2024-03-12T14:30:15Z |
| format | Article |
| id | doaj.art-5a3951849aae46f3a22c1aab3de881fa |
| institution | Directory Open Access Journal |
| issn | 1664-8021 |
| language | English |
| last_indexed | 2024-03-12T14:30:15Z |
| publishDate | 2023-08-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Genetics |
| spelling | doaj.art-5a3951849aae46f3a22c1aab3de881fa2023-08-17T13:30:21ZengFrontiers Media S.A.Frontiers in Genetics1664-80212023-08-011410.3389/fgene.2023.12502761250276Nucleic acid-based therapeutics for the treatment of central nervous system disordersRobyn McCartan0Robyn McCartan1Olga Khorkova2Olga Khorkova3Claude-Henry Volmar4Claude-Henry Volmar5Claes Wahlestedt6Claes Wahlestedt7Center for Therapeutic Innovation, University of Miami Miller School of Medicine, Miami, Florida, United StatesDepartment of Psychiatry and Behavioral Sciences, University of Miami Miller School of Medicine, Miami, Florida, United StatesCenter for Therapeutic Innovation, University of Miami Miller School of Medicine, Miami, Florida, United StatesOPKO Health, Miami, Florida, United StatesCenter for Therapeutic Innovation, University of Miami Miller School of Medicine, Miami, Florida, United StatesDepartment of Psychiatry and Behavioral Sciences, University of Miami Miller School of Medicine, Miami, Florida, United StatesCenter for Therapeutic Innovation, University of Miami Miller School of Medicine, Miami, Florida, United StatesDepartment of Psychiatry and Behavioral Sciences, University of Miami Miller School of Medicine, Miami, Florida, United StatesNucleic acid-based therapeutics (NBTs) are an emerging class of drugs with potential for the treatment of a wide range of central nervous system conditions. To date, pertaining to CNS indications, there are two commercially available NBTs and a large number of ongoing clinical trials. However, these NBTs are applied directly to the brain due to very low blood brain barrier permeability. In this review, we outline recent advances in chemical modifications of NBTs and NBT delivery techniques intended to promote brain exposure, efficacy, and possible future systemic application.https://www.frontiersin.org/articles/10.3389/fgene.2023.1250276/fullsiRNAASOoligonucleotideblood brain barrierdrug discovery |
| spellingShingle | Robyn McCartan Robyn McCartan Olga Khorkova Olga Khorkova Claude-Henry Volmar Claude-Henry Volmar Claes Wahlestedt Claes Wahlestedt Nucleic acid-based therapeutics for the treatment of central nervous system disorders Frontiers in Genetics siRNA ASO oligonucleotide blood brain barrier drug discovery |
| title | Nucleic acid-based therapeutics for the treatment of central nervous system disorders |
| title_full | Nucleic acid-based therapeutics for the treatment of central nervous system disorders |
| title_fullStr | Nucleic acid-based therapeutics for the treatment of central nervous system disorders |
| title_full_unstemmed | Nucleic acid-based therapeutics for the treatment of central nervous system disorders |
| title_short | Nucleic acid-based therapeutics for the treatment of central nervous system disorders |
| title_sort | nucleic acid based therapeutics for the treatment of central nervous system disorders |
| topic | siRNA ASO oligonucleotide blood brain barrier drug discovery |
| url | https://www.frontiersin.org/articles/10.3389/fgene.2023.1250276/full |
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