"Transformative Approaches to Alzheimer Disease Treatment: Current Progress and Future Direction”

Abstract

Alzheimer's disease (AD) is a neurological condition that worsens over time and is characterised by tau tangles and amyloid plaques. Current medications mostly address symptoms, despite significant research advancement, leaving a demand for disease-modifying therapy unfulfilled. New treatment approaches target the genetic and molecular causes of AD by focussing on gene editing, specifically the CRISPR-Cas9 system. By specifically editing genes linked to disease, including APP, PSEN1, PSEN2, and APOE4, CRISPR-Cas9 aims to improve neuronal protection and lessen tau and amyloid-beta pathology. Base and prime editing are two recent developments in CRISPR technology that have increased therapeutic potential, decreased off-target effects, and improved editing specificity. In cellular and animal models, preclinical research shows that it is effective in repairing mutations, modifying gene expression, and lowering the synthesis of neurotoxic proteins. Adeno-associated viruses (AAVs) and lipid nanoparticles are two delivery systems that have been created to get across the blood-brain barrier's obstacles. Clinical use is nevertheless hampered by concerns about long-term safety, distribution efficiency, and ethical issues, despite encouraging results. CRISPR-Cas9's combination with immunotherapy and artificial intelligence has created new opportunities for precision medicine by providing specialised methods to address the pathological and genetic characteristics of AD. With its potential to shift AD treatment from symptomatic management to curative approaches, this review focusses on the advancements, difficulties, and prospects of CRISPR-Cas9 in AD therapies. To advance CRISPR-based medicines into clinical practice, ongoing innovation in gene-editing technologies, delivery methods, and ethical frameworks will be essential