Current neuropharmacologyJan 22, 2026

Using CRISPR to Develop Genetic Approaches for Protecting the Brain in Alzheimer's Disease

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Jan 23, 2026

Abstract

CRISPR/Cas9 gene editing may effectively target genetic drivers of Alzheimer's Disease.

The technology can disrupt pathogenic genetic variants associated with Alzheimer's disease.
It has been shown to correct mutations in stem cell-derived models, normalizing levels of amyloid-beta.
Preclinical evidence suggests potential reversal of amyloid accumulation and synaptic damage.
Key challenges include risks of unintended genetic changes and difficulties in delivering the therapy across the blood-brain barrier.
Future advancements may improve delivery mechanisms and incorporate newer gene-editing techniques.

Simplified

Alzheimer's Disease (AD), the primary etiology of dementia, remains a considerable challenge owing to the limited availability of pharmacological interventions that effectively modify the course of the disease. This review evaluates CRISPR/Cas9 gene editing as a therapeutic strategy for AD, focusing on its capacity to target genetic drivers (e.g., APP, APOE, PSEN1/2, CD2AP) and modify disease pathology. CRISPR offers unprecedented precision in disrupting AD-associated pathogenic alleles, addressing the limitations of conventional Aβ/tau-targeted therapies that have failed in clinical trials. CRISPR corrects mutations in iPSC/organoid models, normalizing Aβ42/40 ratios and reducing tau hyperphosphorylation. Preclinical studies demonstrate reversal of amyloid accumulation and synaptic degeneration. Key challenges include off-target effects, blood-brain barrier (BBB) delivery limitations, and ethical concerns around permanent genome modifications. This study emphasizes that CRISPR/Cas9 holds transformative potential for AD therapy by targeting root genetic causes. Future success hinges on enhancing delivery systems (e.g., BBB-penetrant vectors) and integrating next-generation editors (base/prime editing) for clinical translation.

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Using CRISPR to Develop Genetic Approaches for Protecting the Brain in Alzheimer's Disease

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