U.S., March 5 -- ClinicalTrials.gov registry received information related to the study (NCT06856759) titled 'Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome' on Dec. 29, 2024.
Brief Summary: Rett syndrome (RTT) is a serious neurodevelopmental disorder that has a significant impact on patients and their families. Patients suffer from severe social dysfunction and poor quality of life, and there is currently no effective treatment available. The MECP2 functional loss mutation is the clear pathogenic factor. In recent years, gene therapy has been applied in neuromuscular diseases such as SMA and has achieved good safety and effectiveness. Professor Qiu Zilong's self-developed AAV-MECP2 gene therapy product for RTT was ...