X-reactivation as an approach to treating Rett Syndrome

Tue19  Oct04:30pm(30 mins)
Hall 1B
Prof Jeannie Lee


The human X chromosome harbors only 4% of our genes but over 20% of loci associated with intellectual disability. Because they inherit only one X-chromosome, males are more frequently affected by X-linked neurodevelopmental genetic disorders. Despite inheriting two X-chromosomes, however, females can also be affected because X-chromosome inactivation (XCI) enables only one of two X chromosomes to be expressed per cell. Notable examples include Rett, CDKL5, and Fragile X syndromes. For these disorders, disease-specific treatments have remained elusive. In female subjects, a cure may be found within their own cells, as every sick cell carries a healthy copy of the affected gene on the inactive X (Xi). Selective Xi-reactivation may therefore be a viable approach that would address the root cause of various X-linked disorders. We will discuss current approaches to reactivate the silent MECP2 allele.
To view the video assoicated with this lecture click here

Hosted By

The European Laboratory Research & Innovation Group Our Vision : To provide outstanding, leading edge knowledge to the life sciences community on an open access basis
Event Logo Find us on Facebook Follow us on Twitter

Get the App

Get this event information on your mobile by
going to the appstore or google play and search for 'elrig'
Eventflo Home
copyright ELRIG, eventflo.co.uk, Labhoo Ltd 2003-2021