Overview

Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)

Status:
ENROLLING_BY_INVITATION

Trial end date:
2027-10-01

Target enrollment:

Participant gender:

Summary

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Charcot-Marie-Tooth disease type 2D (CMT2D) due to a pathogenic, de novo deletion mutation in GARS1

Phase:

PHASE1

Details

Lead Sponsor:

n-Lorem Foundation

Collaborator:

The University of Texas Health Science Center, Houston