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New England Southern A Self-Regulating Gene Therapy Vector for Treating Rett Syndrome

JSHS · 2025

Yifan Ding, Zhenghui Li · Boston Latin School, Boston, MA

Overview

Rett syndrome is a devastating neurodevelopmental disorder caused by loss -of-function mutations in the MeCP2 gene. Gene therapy represents one of the most promising strategies for treating or potentially curing this genetic disease. However, a key challenge in developing an effective gene replacement therapy for Rett syndrome is achieving sufficient MeCP2 express ion in affected cells without risking harmful overexpression. The aim of my study is to engineer a gene self-regulation system that maintains proper MeCP2 expression levels necessary for therapeutic benefit, while avoiding the detrimental effects associate d with MeCP2 overexpression. Building on my previous research presented at JSHS, I introduce a novel gene self -regulation system, “TREAD-Dimmer”, which leverages mechanisms of termination readthrough of the stop codon UGA and Cre -mediated recombination. In the TREAD-Dimmer system, high expression of the target gene triggers increased readthrough of a stop codon -containing sequence, leading to the translation of Cre recombinase fused downstream of the target gene. This results in Cre-mediated DNA recombination and self-deletion of the target gene, effectively downregulating its expression. Through cell culture experiments, I validated that TREAD -Dimmer can be applied to self - regulating gene therapy vectors, preventing overexpression of both green fluorescent protein and human MeCP2. Our ongoing collaborative efforts seek to further translate the TREAD -Dimmer system and its associated MeCP2 gene therapy vector into clinical applications. In conclusion, TREAD-Dimmer offers a promising solution to prevent overexpression in gene therapy, including MeCP2 gene replacement therapy for Rett syndrome, potentially improving therapeutic outcomes and minimizing risks associated with gene therapy treatments.

Competition history

  • JSHS 2025 Category not listed

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Source: Junior Science and Humanities Symposium

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