REWRITING AUTISM AT THE GENETIC LEVEL : EMERGING GENE THERAPY STRATEGIES TRANSFORMING AUTISM SPECTRUM DISORDER TREATMENT – A COLLECTIVE REVIEW

Autism Spectrum Disorder (ASD) is a complex neurodevelopmental condition marked by
repetitive behaviors and social communication challenges. While traditional treatments like
behavioral therapy and antipsychotics manage symptoms such as irritability and
hyperactivity, they do not address the underlying biological causes. Recent genomic research
has identified mutations in genes responsible for synaptic development and neuronal
signaling—such as SHANK3, MECP2, FMR1, and CNTNAP2—as primary drivers of the
disorder. The review examines the application of three innovative molecular pillars designed
to target these genetic roots: Viral Vector-Mediated Gene Delivery: Utilizing engineered
adeno-associated viruses (AAVs), specifically serotypes like AAV9, to deliver functional
"replacement" copies of genes across the blood-brain barrier.CRISPR-Cas9 Genome Editing:
Acting as "molecular scissors," this complex uses a guide RNA to physically cut and
permanently repair DNA mutations or reactivate silenced genes.Antisense Oligonucleotides
(ASOs): Functioning as a "genetic dimmer switch," these synthetic strands target mRNA to
silence faulty genes or modify protein splicing without permanently changing the host
genome.Improved synaptic communication between neurons.
Restoration of neuronal network function. A measurable reduction in autism-like behavioral
abnormalities. The potential for long-lasting, disease-modifying outcomes rather than
lifelong symptom management.
Gene therapy represents a paradigm shift toward precision medicine for ASD. While the
potential for reversing core deficits is high, significant hurdles remain, including ensuring the
safety of brain-wide delivery and minimizing off-target genetic effects. Future progress
depends on advancing early genetic diagnosis and conducting rigorous clinical trials to
transition these "molecular tools" into standard clinical practice.