There was a mistake in the caption of figure 1C as published. "(C) Preclinical efficacy: In MECP2-null mice, scAAV-miniMECP2 improved neurological function, breathing, dendritic spine density, and survival without overexpression toxicity. miRARE restricted expression in wild-type neurons (~8%) but expanded it in deficient neurons (~40%), providing therapeutic benefit."The corrected caption of figure 1C appears below."(C) Preclinical efficacy: In MECP2-null mice, scAAV-miniMECP2 improved neurological function, breathing, and survival without overexpression toxicity."The original version of this article has been updated.Adding/removing text Treated animals demonstrated significant improvements in neurological function, normalized breathing patterns, extended survival (from ~10 weeks to >1 year), and partial restoration of dendritic spine density-all without the toxicity associated with full-length MECP2 overexpression in wild-type mice.
Debopam Samanta (Mon,) studied this question.