Story perspectives

Breakthrough Gene Therapy for Angelman Syndrome Enters Trials

9/19/2025

Summary

Story summary

UC Davis researchers are developing a gene therapy for Angelman syndrome, affecting 1 in 15,000 births.
The therapy aims to replace a missing protein due to the loss of the UBE3A gene, potentially reversing symptoms.
Funded by a $4.5 million grant, the project is led by Jill Silverman and Joseph Anderson.
Initial studies in mouse models showed success, challenging previous treatment limitations.
The focus is now on safety, with plans for human clinical trials.

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Story summary

UC Davis researchers are developing a gene therapy for Angelman syndrome, affecting 1 in 15,000 births.
The therapy aims to replace a missing protein due to the loss of the UBE3A gene, potentially reversing symptoms.
Funded by a $4.5 million grant, the project is led by Jill Silverman and Joseph Anderson.
Initial studies in mouse models showed success, challenging previous treatment limitations.
The focus is now on safety, with plans for human clinical trials.