Personalized Antisense Oligonucleotide Therapy for a Single Participant with ATN1 Gene Mutation
Status:
ACTIVE_NOT_RECRUITING
Trial end date:
2026-02-01
Target enrollment:
Participant gender:
Summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with dentatorubral-pallidoluysian atrophy (DRPLA) due to a heterozygous pathogenic CAG trinucleotide expansion in ATN1