A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)
Conditions
- MECP2 Duplication Syndrome
Interventions
Sponsor
HuidaGene Therapeutics Co., Ltd.
Collaborators