Oligonucleotides as a Novel Therapeutic Approach: An Innovative Area for Drug Delivery in Neurological Disorders
RNA-based therapeutics have emerged as one of the most potent therapeutic options used for the modulation of gene/protein expression and gene editing with the potential to treat neurodegenerative diseases. However, the delivery of nucleic acids to the central nervous system (CNS) by the systemic ro...
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| Format: | Article |
| Language: | English |
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College of Pharmacy / Mustansiriyah University
2025-01-01
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| Series: | Al-Mustansiriyah Journal of Pharmaceutical Sciences |
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| Online Access: | https://ajps.uomustansiriyah.edu.iq/index.php/AJPS/article/view/1065 |
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| author | Yasir Qasim Almajidi Rana Kadum Muslim Yaseen T. khalf |
| author_facet | Yasir Qasim Almajidi Rana Kadum Muslim Yaseen T. khalf |
| author_sort | Yasir Qasim Almajidi |
| collection | DOAJ |
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RNA-based therapeutics have emerged as one of the most potent therapeutic options used for the modulation of gene/protein expression and gene editing with the potential to treat neurodegenerative diseases. However, the delivery of nucleic acids to the central nervous system (CNS) by the systemic route, remains a major hurdle.
To overcome this pitfall, this review focuses on oligonucleotide-based novel strategies including liposomes, carbon nanotubes, quantum dots, solid lipid nanoparticles, nano lipid carriers, polymeric nanoparticles, mesoporous silica, dendrimers, aptamers, nanobodies etc. These strategies are designed to overcome these barriers by different pathways and mechanisms of transport across the blood−brain barrier. Ongoing preclinical and clinical studies are assessing the safety and efficacy of Antisense Oligonucleotide) ASOs (in multiple genetic and acquired neurological conditions. The current review provides an update on novel approaches, preclinical, clinical evidence, and delivery route of ASOs. The administration of FDA-approved ASOs in neurological disorders is also described. The current evidence on the safety and efficacy of ASOs in brain diseases will help identify opportunities for a broader range of nucleic acids and accelerate the clinical translation of these innovative therapies.
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| format | Article |
| id | doaj-art-60c2db2c486d4f40b6535db80352224e |
| institution | Kabale University |
| issn | 1815-0993 2959-183X |
| language | English |
| publishDate | 2025-01-01 |
| publisher | College of Pharmacy / Mustansiriyah University |
| record_format | Article |
| series | Al-Mustansiriyah Journal of Pharmaceutical Sciences |
| spelling | doaj-art-60c2db2c486d4f40b6535db80352224e2025-01-24T13:47:28ZengCollege of Pharmacy / Mustansiriyah UniversityAl-Mustansiriyah Journal of Pharmaceutical Sciences1815-09932959-183X2025-01-0125110.32947/ajps.v25i1.1065Oligonucleotides as a Novel Therapeutic Approach: An Innovative Area for Drug Delivery in Neurological DisordersYasir Qasim Almajidi0Rana Kadum Muslim1Yaseen T. khalf 2Department of pharmacy, Baghdad College of Medical Sciences, Baghdad, IraqDepartment of pharmacy, Baghdad College of Medical Sciences, Baghdad, IraqDepartment of pharmacy, Alfarabi University College RNA-based therapeutics have emerged as one of the most potent therapeutic options used for the modulation of gene/protein expression and gene editing with the potential to treat neurodegenerative diseases. However, the delivery of nucleic acids to the central nervous system (CNS) by the systemic route, remains a major hurdle. To overcome this pitfall, this review focuses on oligonucleotide-based novel strategies including liposomes, carbon nanotubes, quantum dots, solid lipid nanoparticles, nano lipid carriers, polymeric nanoparticles, mesoporous silica, dendrimers, aptamers, nanobodies etc. These strategies are designed to overcome these barriers by different pathways and mechanisms of transport across the blood−brain barrier. Ongoing preclinical and clinical studies are assessing the safety and efficacy of Antisense Oligonucleotide) ASOs (in multiple genetic and acquired neurological conditions. The current review provides an update on novel approaches, preclinical, clinical evidence, and delivery route of ASOs. The administration of FDA-approved ASOs in neurological disorders is also described. The current evidence on the safety and efficacy of ASOs in brain diseases will help identify opportunities for a broader range of nucleic acids and accelerate the clinical translation of these innovative therapies. https://ajps.uomustansiriyah.edu.iq/index.php/AJPS/article/view/1065Antisense OligonucleotideNeurodegenerativeSmall interfering RNAMicro RNABlood−brain barrierTherapeutic responses |
| spellingShingle | Yasir Qasim Almajidi Rana Kadum Muslim Yaseen T. khalf Oligonucleotides as a Novel Therapeutic Approach: An Innovative Area for Drug Delivery in Neurological Disorders Al-Mustansiriyah Journal of Pharmaceutical Sciences Antisense Oligonucleotide Neurodegenerative Small interfering RNA Micro RNA Blood−brain barrier Therapeutic responses |
| title | Oligonucleotides as a Novel Therapeutic Approach: An Innovative Area for Drug Delivery in Neurological Disorders |
| title_full | Oligonucleotides as a Novel Therapeutic Approach: An Innovative Area for Drug Delivery in Neurological Disorders |
| title_fullStr | Oligonucleotides as a Novel Therapeutic Approach: An Innovative Area for Drug Delivery in Neurological Disorders |
| title_full_unstemmed | Oligonucleotides as a Novel Therapeutic Approach: An Innovative Area for Drug Delivery in Neurological Disorders |
| title_short | Oligonucleotides as a Novel Therapeutic Approach: An Innovative Area for Drug Delivery in Neurological Disorders |
| title_sort | oligonucleotides as a novel therapeutic approach an innovative area for drug delivery in neurological disorders |
| topic | Antisense Oligonucleotide Neurodegenerative Small interfering RNA Micro RNA Blood−brain barrier Therapeutic responses |
| url | https://ajps.uomustansiriyah.edu.iq/index.php/AJPS/article/view/1065 |
| work_keys_str_mv | AT yasirqasimalmajidi oligonucleotidesasanoveltherapeuticapproachaninnovativeareafordrugdeliveryinneurologicaldisorders AT ranakadummuslim oligonucleotidesasanoveltherapeuticapproachaninnovativeareafordrugdeliveryinneurologicaldisorders AT yaseentkhalf oligonucleotidesasanoveltherapeuticapproachaninnovativeareafordrugdeliveryinneurologicaldisorders |