Life sciences · Journal article
Astana Medical Journal · October 23, 2026
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Alzheimer’s disease (AD) is the leading cause of neurodegenerative dementia and represents a significant worldwide health burden. The disease is marked by a gradual deterioration of cognitive abilities, which is closely linked to the accumulation of amyloid-β (Aβ), the development of tau-related pathology, impaired synaptic function, and progressive neuronal degeneration. Although substantial advances have been made in understanding AD, the therapies currently available primarily offer modest symptomatic relief and have limited effectiveness in slowing or altering the underlying progression of the disease. Consequently, novel therapeutic strategies that target the underlying molecular mechanisms of AD are urgently needed. This review analyzes recent scientific literature on the application of CRISPR/Cas9 genome-editing technology in AD research and therapy. The literature was selected based on its relevance to AD-associated genetic targets, genome-editing strategies, delivery systems, therapeutic outcomes, and safety considerations, with particular attention to preclinical studies involving APP, PSEN1, PSEN2, APOE, BACE1, and SORL1. This review examines the emerging potential of CRISPR/Cas9 genome-editing technology as a therapeutic approach for Alzheimer's disease. CRISPR-based strategies enable precise modification of genes implicated in AD pathogenesis, including APP, PSEN1, PSEN2, and APOE, with the aim of reducing amyloidogenic processing, correcting pathogenic mutations, or modifying genetic risk factors. Recent preclinical studies have demonstrated encouraging results, including reduced amyloid pathology, improved neuronal function, and enhanced cognitive performance in experimental models. We also discuss current delivery approaches, including viral and non-viral systems, and summarize recent advances in genome-editing platforms that may improve therapeutic precision and safety. Despite its significant therapeutic potential, the clinical application of CRISPR/Cas9 remains constrained by several important obstacles. These include the difficulty of achieving effective delivery to the brain across the blood–brain barrier, the possibility of unintended genomic modifications, activation of immune reactions, and uncertainties regarding the long-term safety and consequences of genome editing. Continued improvements in editing accuracy, delivery technologies, and comprehensive preclinical evaluation will be essential before CRISPR-based therapies can become a viable treatment option for Alzheimer's disease. Keywords: Alzheimer’s disease, CRISPR/Cas9, gene editing, neurodegeneration, APOE4, amyloid-beta.