Gene Therapy as a Novel Strategy for Alzheimer’s Disease: Efficacy and Long-Term Implications
Jeetendra Kumar Guptaa, Kuldeep Singha✉, Divya Jainb, Alok Bhattc, Anubhav Dubeyd, Mukesh Chandra Sharmae, Mohammad Tabishf g, Pranav Sharmah, Abdullah Al Nomani
- aDepartment of Pharmacology, Institute of Pharmaceutical Research, GLA University, Mathura, Uttar Pradesh, India
- bDepartment of Microbiology, School of Applied and Life Sciences, Uttaranchal university, Dehradun-248007, Uttarakhand,India
- cSchool of Pharmacy, Graphic Era Hill University, Bell Road, Clement Town, Dehradun, Uttarakhand,India
- dDepartment of Pharmacology Maharana Pratap College of Pharmacy kanpur Uttar Pradesh India
- eSchool of Pharmacy, Devi Ahilya Vishwavidalaya, Indore, M.P, India
- fCentre for Global Health Research, Saveetha Medical College and Hospital, Saveetha Institute of Medical and Technical Sciences (SIMATS), Saveetha University, Chennai, Tamil Nadu, India
- gDepartment of Pharmacology, College of Medicine, Shaqra University, Shaqra 11961, Saudi Arabia
- hBiotechnology Program, Department of Mathematics and Natural Science, BRAC University, Dhaka,Bangladesh
- iSchool of Pharmacy, BRAC University, Dhaka, Bangladesh
- ✉Corresponding author
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Abstract
Abstract: Alzheimer's disease (AD) represents a significant healthcare challenge, requiring innovative treatment strategies. This review examines gene therapy as a promising approach for targeting fundamental biological pathways involved in the development and progression of AD. Focusing on the genetic and molecular underpinnings of the disease, the paper highlights pathological indicators such as beta-amyloid plaques and neurofibrillary tangles. It explores the rationale for gene therapy, emphasizing the manipulation of genes implicated in amyloid processing, tau protein regulation, and neuroinflammation. The analysis critically evaluates the effectiveness of various gene therapy interventions in mitigating cognitive decline, reducing neurodegeneration, and altering disease progression. Furthermore, the long-term safety profiles and potential adverse effects associated with gene therapies are discussed, including immune responses and therapeutic limitations. The conclusion synthesizes current findings, identifies research gaps, and proposes future directions for the advancement of gene therapy as a viable and sustainable strategy for treating Alzheimer's disease.
TL;DRThis review examines gene therapy as a promising approach for targeting fundamental biological pathways involved in the development and progression of AD, exploring the rationale for gene therapy and the manipulation of genes implicated in amyloid processing, tau protein regulation, and neuroinflammation. (Semantic Scholar)

