Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A
Status:
ACTIVE_NOT_RECRUITING
Trial end date:
2026-02-16
Target enrollment:
Participant gender:
Summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single pediatric participant with SCN2A associated developmental epileptic encephalopathy
Phase:
PHASE1
Details
Lead Sponsor:
University of California, San Diego
Collaborators:
California Institute for Regenerative Medicine (CIRM) n-Lorem Foundation