Alzheimer’s disease: A CRISPR/CAS9-mediated therapeutic approach
1 Sardar Patel College of Pharmacy, Medical College Road, Gorakhpur 273013, (U.P.), India.
2 CSIR-Central Institute of Medicinal and Aromatic Plants, Lucknow 226015. (U.P), India.
3 Integral University, Kursi Road, Lucknow 226015. (U.P), India.
4 Faculty of Pharmaceutical Sciences, Mahayogi Gorakhnath University, Gorakhpur 273007 (U.P.), India
5 Dr. Mahendra Kumar Chhote Lal Bind college of Pharmacy Upardaha handia Prayagraj, (U.P.), India.
Review
World Journal of Biology Pharmacy and Health Sciences, 2024, 19(02), 035–049.
Article DOI: 10.30574/wjbphs.2024.19.2.0493
Publication history:
Received on 23 June 2024; revised on 30 July 2024; accepted on 02 August 2024
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.
Keywords:
CRISPR/Cas9; Alzheimer’s Disease; APP; APOE; PSEN1; PSEN2; Amyloid beta; Tau protein
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