Alzheimer’s disease: A CRISPR/CAS9-mediated therapeutic approach

Abstract

The degenerative nature of Alzheimer's disease (AD) and its severe effects on cognitive function present a major challenge to worldwide healthcare systems. CRISPR/Cas9, one of the most recent developments in gene-editing technology, has created new opportunities to investigate possible AD treatment approaches. The present state of AD research is reviewed in this paper, along with the possibility of using CRISPR/Cas9-mediated methods to target important genetic elements involved in AD pathogenesis. Through targeted gene editing linked to tau protein malfunction, neuroinflammation, and amyloid-beta accumulation, CRISPR/Cas9 presents a viable approach to altering the molecular course of disease. Additionally, using CRISPR/Cas9 in patient-specific induced pluripotent stem cells (iPSCs) may lead to personalized medicine strategies for the treatment of AD. Issues like delivery strategies, off-target impacts, and moral dilemmas are also covered. All things considered, the application of CRISPR/Cas9 technology to AD research is a fresh and potentially revolutionary strategy for creating targeted treatments for this intricate neurodegenerative illness. For the purpose of treating AD, more preclinical and clinical research is necessary to confirm the security and effectiveness of CRISPR/Cas9-based therapies. With the use of the CRISPR/Cas9 system, precise and effective genome modification is possible, enabling targeted editing of particular genes linked to the pathophysiology of AD. Thanks to this technology, genetic mutations in the presenilin 1 (PSEN1), presenilin 2 (PSEN2), and amyloid precursor protein (APP) genes that are linked to family types of AD can be corrected. It is feasible to restore normal protein function and possibly lessen the pathogenic processes that underlie AD by fixing these mutations.