Gene Editing in Neurological Disorders: Role of CRISPR-Cas9 in Targeted Therapies

Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, and amyotrophic lateral sclerosis are complex neurological disorders driven by intricate genetic and molecular mechanisms. Current therapies are mainly symptomatic and fail to correct underlying genetic defects. The advent of CRISPR-Cas9 gene-editing technology offers unprecedented opportunities to address these disorders at the genomic level through precise DNA editing, gene regulation, and epigenetic modulation. Preclinical studies using neuronal models, brain organoids, and transgenic animals have demonstrated the feasibility of correcting gene defects with CRISPR. Advances such as improved delivery systems, high-fidelity Cas9 variants, and base editing further strengthen its clinical potential. However, key challenges remain, including safe delivery across the blood–brain barrier, minimizing off-target effects, and addressing ethical concerns. Overall, CRISPR-Cas9 holds promise for integration into precision medicine to revolutionize treatment of neurodegenerative and neurodevelopmental diseases.