CRISPR/Cas9-Based therapeutics as a promising strategy for management of Alzheimer’s disease: progress and prospects DOI Creative Commons

Mohamad Sultan Khan,

Nousheen Qureshi,

Rehan Khan

и другие.

Frontiers in Cellular Neuroscience, Год журнала: 2025, Номер 19

Опубликована: Апрель 7, 2025

CRISPR/Cas9 technology has revolutionized genetic and biomedical research in recent years. It enables editing modulation of gene function with an unparalleled precision effectiveness. Among the various applications prospects this technology, opportunities it offers unraveling molecular underpinnings a myriad central nervous system diseases, including neurodegenerative disorders, psychiatric conditions, developmental abnormalities, are unprecedented. In review, we highlight CRISPR/Cas9-based therapeutics as promising strategy for management Alzheimer's disease transformative impact on AD research. Further, emphasize role generating accurate models identification novel therapeutic targets, besides CRISPR-based therapies aimed at correcting AD-associated mutations modulating processes. Furthermore, delivery systems reviewed potential non-viral nanotechnology-based carriers overcoming critical limitations effective is discussed. Overall, review highlights promise intricate processes underlying development AD, discusses its limitations, ethical concerns several challenges efficient across BBB, ensuring specificity, avoiding off-target effects. This article can be helpful better understanding based approaches way forward utilizing enormous targeted, gene-specific treatments that could change trajectory debilitating incurable illness.

Язык: Английский

CRISPR/Cas9-Based therapeutics as a promising strategy for management of Alzheimer’s disease: progress and prospects DOI Creative Commons

Mohamad Sultan Khan,

Nousheen Qureshi,

Rehan Khan

и другие.

Frontiers in Cellular Neuroscience, Год журнала: 2025, Номер 19

Опубликована: Апрель 7, 2025

CRISPR/Cas9 technology has revolutionized genetic and biomedical research in recent years. It enables editing modulation of gene function with an unparalleled precision effectiveness. Among the various applications prospects this technology, opportunities it offers unraveling molecular underpinnings a myriad central nervous system diseases, including neurodegenerative disorders, psychiatric conditions, developmental abnormalities, are unprecedented. In review, we highlight CRISPR/Cas9-based therapeutics as promising strategy for management Alzheimer's disease transformative impact on AD research. Further, emphasize role generating accurate models identification novel therapeutic targets, besides CRISPR-based therapies aimed at correcting AD-associated mutations modulating processes. Furthermore, delivery systems reviewed potential non-viral nanotechnology-based carriers overcoming critical limitations effective is discussed. Overall, review highlights promise intricate processes underlying development AD, discusses its limitations, ethical concerns several challenges efficient across BBB, ensuring specificity, avoiding off-target effects. This article can be helpful better understanding based approaches way forward utilizing enormous targeted, gene-specific treatments that could change trajectory debilitating incurable illness.

Язык: Английский

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