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Spotlighting Rare Diseases Research Blog

Optimizing Genome Editing Technologies for Therapeutic Application

Posted On: Jul 16, 2019


The research in my laboratory is focused on improving genome editing technologies so that they can be applied to the development of therapeutics. Inspired by the opportunity to improve the lives of patients with debilitating disorders, my lifelong goal as a scientist is to translate genome editing technologies to the development of therapeutics for a variety of diseases, both common and rare.


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Reactivating the Silenced X Chromosome: A Potential Therapeutic Approach to CDKL5 Deficiency and Rett Syndrome

Posted On: Jun 6, 2019


Several years ago, we began investigating the mechanism by which genes are turned off on the X chromosome — a process termed X chromosome inactivation (XCI). In females, XCI is a naturally occurring phenomenon in which one of the X chromosomes is randomly turned off, or silenced, as a way to equalize the dosage of the X chromosome between females (who have two X chromosomes) and males (who have one).

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