CRISPR’s Promise for Rett Syndrome and Beyond

Unlocking New Genetic Therapies for a Rare and Devastating Disorder

Abstract

Rett Syndrome is a rare neurodevelopmental disorder that primarily affects girls and is caused by mutations in the MECP2 gene on the X chromosome. Current treatments focus on managing symptoms, but they don’t address the underlying genetic cause. CRISPR gene editing offers a promising way to directly fix the faulty gene responsible for the disorder. New techniques, such as base and prime editing, provide precise and safer corrections, while X-chromosome reactivation may help females by activating their healthy copy of MECP2. Delivering these therapies across the blood-brain barrier remains a significant challenge, but advances using viral vectors, nanoparticles, and stem cell methods are encouraging. Combining CRISPR with existing drugs or RNA editing could further improve outcomes, especially for patients who have already developed symptoms. Although still in early stages, CRISPR-based approaches provide hope for treating Rett Syndrome at its root, and open doors for future therapies for other rare genetic brain disorders.