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Gene Therapy Reverses SRD Symptoms in Mice: Breakthrough!

10/10/2025

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Summary

Story summary

Allen Institute researchers developed a gene therapy reversing SRD symptoms in mice.
In Molecular Therapy, the therapy used an adeno-associated virus (AAV) to deliver a functional SYNGAP1 gene.
It reduced epileptic activity, normalized brain waves, and lowered hyperactivity and risk-taking.
Treatment was effective at neonatal and juvenile stages.
Future refinements could target specific neuron types to improve safety and efficacy.

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Story summary

Allen Institute researchers developed a gene therapy reversing SRD symptoms in mice.
In Molecular Therapy, the therapy used an adeno-associated virus (AAV) to deliver a functional SYNGAP1 gene.
It reduced epileptic activity, normalized brain waves, and lowered hyperactivity and risk-taking.
Treatment was effective at neonatal and juvenile stages.
Future refinements could target specific neuron types to improve safety and efficacy.