CRISPR Therapeutics in Neurology and Neurosurgery
Abstract
Abstract Background: CRISPR platforms could modify molecular causes of neurological disease and engineer cells used in neurosurgical treatment. Their clinical value depends on disease mechanism, editing modality, anatomical delivery and the capacity of surviving neural circuits to recover.Objective: To assess CRISPR therapeutics in neurology and neurosurgery, distinguish human evidence from preclinical findings, and identify requirements for clinical translation.Methods: A targeted narrative review of web-indexed PubMed records, primary publications, trial descriptions, regulatory guidance and sponsor reports was conducted through 11 October 2026. Evidence was assessed by study design, model, delivery, functional outcomes and source maturity. No meta-analysis or formal certainty grading was undertaken.Findings: Human evidence includes hepatic editing for hereditary transthyretin amyloid polyneuropathy and edited immune cells for high-grade glioma. RNA targeting for MECP2 duplication syndrome and epigenetic treatment for facioscapulohumeral muscular dystrophy have entered human investigation. Preclinical studies support mutation correction, dosage restoration, toxic-product suppression and regenerative cell engineering. Constraints include target-cell delivery, unintended genomic changes, dosage-dependent toxicity, immunity, procedural injury and neural loss. Avoiding DNA cleavage does not eliminate biological or delivery-related risks.Conclusions: CRISPR is a credible investigational platform for selected neurological and neurosurgical indications. Development should prioritize defined disease mechanisms, measurable target engagement, relevant tissue coverage and a defensible safety margin. Routine adoption requires comparative clinical evidence and sustained surveillance. Neurosurgery provides anatomical access and tissue-based assessment, but procedural feasibility alone cannot establish benefit. Keywords: CRISPR-Cas systems; genome editing; neurology; neurosurgery; neurodegenerative diseases; brain neoplasms; gene therapy
