Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome

"For Dose 1, the first 5 subjects will be enrolled in the trial in sequence, with one patient completing the administration and no significant dose limiting toxicity (DLT) observed during a one month follow-up. The latter subject will be enrolled in the trial medication.~Once the fifth subject in Dose 1 completes the administration and no significant DLT is observed after a follow-up period of at least two weeks, the study dose can be escalated to a higher level.~For Dose 2, the 3 subjects will be enrolled in the trial in sequence, with one patient completing the administration and no significant DLT observed during a one month follow-up. The latter subject will be enrolled in the trial medication.~DLT definition: see Study Description."