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UNC Health Advances Gene Therapy Research for Rett Syndrome
UNC Health is progressing gene therapy research aimed at treating Rett syndrome, a rare neurological disorder that primarily affects girls and severely impairs movement, communication and daily function. The development offers hope to families affected by the condition, as gene therapy approaches seek to address the underlying genetic cause rather than only managing symptoms.
Why now: Families and researchers affected by Rett syndrome are following progress toward a potential gene therapy treatment.
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