Gene therapy for autism is still in the early research stages, primarily targeting specific genetic mutations, such as SHANK3, which is associated with autism-like symptoms seen in conditions like Phelan-McDermid syndrome. Researchers are exploring how to target genes involved in brain development and synaptic function to manage or reduce certain symptoms of autism. However, due to the complexity of autism—affected by a multitude of genetic and environmental factors—gene therapy is not currently regarded as a complete cure. Instead, it holds potential for addressing specific genetic forms of the disorder in the future.
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