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Mediterranean Journal of Medicine and Medical Sciences
https://mmj.org.ly/article/6a2b2feba953951216563226

Mediterranean Journal of Medicine and Medical Sciences

Review Molecular biology

Autism in the era of gene editing: A CRISPR-Cas9 review

N Arjun, Ramdas Bhat

Mediterr J Med Med Sci, vol.2, n2, p.96-108, 2026
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Abstract

Autism spectrum disorder (ASD) is a clinically and biologically heterogeneous group of neuro-developmental conditions characterized by differences in social communication and interaction, restricted interests, repetitive behaviors, and variable patterns of sensory processing. Genetic studies have established ASD as one of the most heritable neurodevelopmental conditions. Still, its architecture ranges from rare, highly penetrant monogenic disorders to a broadly polygenic background shaped by thousands of common variants. The development of clustered regularly interspaced short palindromic repeats-associated protein 9 (CRISPR-Cas9) has transformed autism research by enabling rapid, programmable, and scalable interrogation of ASD-associated genes in cellular, organoid, and animal systems. This review synthesizes current CRISPR-Cas9 applications in ASD research, focusing on disease modelling, gene function, next-generation editing technologies, delivery to the central nervous system, safety, clinical translation, and ethics. CRISPR has accelerated the modelling of high-confidence ASD-associated genes, including SHANK3, MECP2, FMR1, CHD8, CNTNAP2, SCN2A, and MEF2C. Recent advances in base editing, prime editing, CRISPR activation, and epigenome editing offer more precise approaches for specific monogenic neurodevelopmental disorders. However, the translation of genome editing into ASD therapy remains constrained by technical barriers, including delivery across the blood-brain barrier, off-target editing, structural rearrangements, mosaicism, immune responses, and developmental timing. More fundamentally, attempts to edit “autism” as a trait raise profound ethical concerns, particularly from neurodiversity and disability-rights perspectives. We argue that the most defensible clinical horizon for CRISPR in this field is not the elimination of autism, but carefully governed somatic intervention for severe, medically disabling features of specific monogenic syndromes, pursued in partnership with autistic people, families, clinicians, and disability communities.

Keywords

Autism spectrum disorder, bioethics, CRISPR-Cas9, gene therapy, genome editing, neurodiversity 

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Submitted date:
05/22/2026

Reviewed date:
06/20/2026

Accepted date:
06/29/2026

Publication date:
06/11/2026

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