CRISPR/Cas9-Based therapeutics as a promising strategy for management of Alzheimer’s disease: progress and prospects
CRISPR/Cas9 technology has revolutionized genetic and biomedical research in recent years. It enables editing and modulation of gene function with an unparalleled precision and effectiveness. Among the various applications and prospects of this technology, the opportunities it offers in unraveling t...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2025-04-01
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| Series: | Frontiers in Cellular Neuroscience |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fncel.2025.1578138/full |
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| author | Mohamad Sultan Khan Nousheen Qureshi Rehan Khan Young-Ok Son Tariq Maqbool |
| author_facet | Mohamad Sultan Khan Nousheen Qureshi Rehan Khan Young-Ok Son Tariq Maqbool |
| author_sort | Mohamad Sultan Khan |
| collection | DOAJ |
| description | CRISPR/Cas9 technology has revolutionized genetic and biomedical research in recent years. It enables editing and modulation of gene function with an unparalleled precision and effectiveness. Among the various applications and prospects of this technology, the opportunities it offers in unraveling the molecular underpinnings of a myriad of central nervous system diseases, including neurodegenerative disorders, psychiatric conditions, and developmental abnormalities, are unprecedented. In this review, we highlight the applications of CRISPR/Cas9-based therapeutics as a promising strategy for management of Alzheimer’s disease and transformative impact of this technology on AD research. Further, we emphasize the role of CRISPR/Cas9 in generating accurate AD models for identification of novel therapeutic targets, besides the role of CRISPR-based therapies aimed at correcting AD-associated mutations and modulating the neurodegenerative processes. Furthermore, various delivery systems are reviewed and potential of the non-viral nanotechnology-based carriers for overcoming the critical limitations of effective delivery systems for CRISPR/Cas9 is discussed. Overall, this review highlights the promise and prospects of CRISPR/Cas9 technology for unraveling the intricate molecular processes underlying the development of AD, discusses its limitations, ethical concerns and several challenges including efficient delivery across the BBB, ensuring specificity, avoiding off-target effects. This article can be helpful in better understanding the applications of CRISPR/Cas9 based therapeutic approaches and the way forward utilizing enormous potential of this technology in targeted, gene-specific treatments that could change the trajectory of this debilitating and incurable illness. |
| format | Article |
| id | doaj-art-eabcb204dfb74bf19fbbd825fe5ea81b |
| institution | OA Journals |
| issn | 1662-5102 |
| language | English |
| publishDate | 2025-04-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Cellular Neuroscience |
| spelling | doaj-art-eabcb204dfb74bf19fbbd825fe5ea81b2025-08-20T02:07:59ZengFrontiers Media S.A.Frontiers in Cellular Neuroscience1662-51022025-04-011910.3389/fncel.2025.15781381578138CRISPR/Cas9-Based therapeutics as a promising strategy for management of Alzheimer’s disease: progress and prospectsMohamad Sultan Khan0Nousheen Qureshi1Rehan Khan2Young-Ok Son3Tariq Maqbool4Laboratory of Nanotherapeutics and Regenerative Medicine, Department of Nanotechnology, University of Kashmir, Srinagar, IndiaDepartment of Higher Education, Government of Jammu and Kashmir, Srinagar, IndiaChemical Biology Unit, Institute of Nano Science and Technology, Knowledge City, Mohali, Punjab, IndiaDepartment of Animal Biotechnology, Faculty of Biotechnology, College of Applied Life Sciences and Interdisciplinary Graduate Program in Advanced Convergence Technology and Science, Jeju National University, Jeju, Republic of KoreaLaboratory of Nanotherapeutics and Regenerative Medicine, Department of Nanotechnology, University of Kashmir, Srinagar, IndiaCRISPR/Cas9 technology has revolutionized genetic and biomedical research in recent years. It enables editing and modulation of gene function with an unparalleled precision and effectiveness. Among the various applications and prospects of this technology, the opportunities it offers in unraveling the molecular underpinnings of a myriad of central nervous system diseases, including neurodegenerative disorders, psychiatric conditions, and developmental abnormalities, are unprecedented. In this review, we highlight the applications of CRISPR/Cas9-based therapeutics as a promising strategy for management of Alzheimer’s disease and transformative impact of this technology on AD research. Further, we emphasize the role of CRISPR/Cas9 in generating accurate AD models for identification of novel therapeutic targets, besides the role of CRISPR-based therapies aimed at correcting AD-associated mutations and modulating the neurodegenerative processes. Furthermore, various delivery systems are reviewed and potential of the non-viral nanotechnology-based carriers for overcoming the critical limitations of effective delivery systems for CRISPR/Cas9 is discussed. Overall, this review highlights the promise and prospects of CRISPR/Cas9 technology for unraveling the intricate molecular processes underlying the development of AD, discusses its limitations, ethical concerns and several challenges including efficient delivery across the BBB, ensuring specificity, avoiding off-target effects. This article can be helpful in better understanding the applications of CRISPR/Cas9 based therapeutic approaches and the way forward utilizing enormous potential of this technology in targeted, gene-specific treatments that could change the trajectory of this debilitating and incurable illness.https://www.frontiersin.org/articles/10.3389/fncel.2025.1578138/fullCNSAlzheimer’s diseasegene editing (CRISPR/Cas9)therapeuticsnanocarriers |
| spellingShingle | Mohamad Sultan Khan Nousheen Qureshi Rehan Khan Young-Ok Son Tariq Maqbool CRISPR/Cas9-Based therapeutics as a promising strategy for management of Alzheimer’s disease: progress and prospects Frontiers in Cellular Neuroscience CNS Alzheimer’s disease gene editing (CRISPR/Cas9) therapeutics nanocarriers |
| title | CRISPR/Cas9-Based therapeutics as a promising strategy for management of Alzheimer’s disease: progress and prospects |
| title_full | CRISPR/Cas9-Based therapeutics as a promising strategy for management of Alzheimer’s disease: progress and prospects |
| title_fullStr | CRISPR/Cas9-Based therapeutics as a promising strategy for management of Alzheimer’s disease: progress and prospects |
| title_full_unstemmed | CRISPR/Cas9-Based therapeutics as a promising strategy for management of Alzheimer’s disease: progress and prospects |
| title_short | CRISPR/Cas9-Based therapeutics as a promising strategy for management of Alzheimer’s disease: progress and prospects |
| title_sort | crispr cas9 based therapeutics as a promising strategy for management of alzheimer s disease progress and prospects |
| topic | CNS Alzheimer’s disease gene editing (CRISPR/Cas9) therapeutics nanocarriers |
| url | https://www.frontiersin.org/articles/10.3389/fncel.2025.1578138/full |
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