Autism spectrum disorder: prospects for treatment using gene therapy

Area of Science:

• Neuroscience
• Genetics
• Developmental Biology

Background:

• Autism spectrum disorder (ASD) prevalence is increasing, with diagnoses in 1 in 59 children.
• ASD is characterized by social interaction impairments, restricted behaviors, and communication difficulties.
• Genetic factors are primary drivers of ASD, suggesting potential for gene therapy.

Purpose of the Study:

• To review the potential of gene therapy as a disease-modifying treatment for ASD.
• To explore advances in modeling ASD and gene delivery to the central nervous system (CNS).
• To assess the applicability of gene therapy for both monogenic and polygenic forms of ASD.

Main Methods:

• Review of preclinical studies in animal models of monogenic ASD.
• Analysis of gene replacement and gene silencing strategies.
• Examination of gene therapy's potential for polygenic ASD through understanding dysregulated pathways.

Main Results:

• Preclinical studies demonstrate feasibility of gene therapy for monogenic ASD.
• Advances in CNS gene delivery are crucial for therapeutic development.
• Understanding common pathways broadens gene therapy scope to polygenic ASD.

Conclusions:

• Gene therapy presents a significant opportunity to address cognitive dysfunction in ASD.
• Despite challenges, gene therapy holds promise for a disease-modifying treatment.
• Further research and development are essential for clinical translation.