Gene editing to treat expanded CAG/CTG repeat disorders

Tue19  Oct03:45pm(30 mins)
Where:
Hall 1C
Speaker:
 Vincent  Dion

Abstract

Expanded CAG/CTG repeats, including Huntington’s disease, myotonic dystrophy, and several spinocerebellar ataxias, remain without a disease altering treatment, which represents an important unmet medical need. Although the clinical manifestation varies greatly between these diseases, their underlying cause is the same: the expansion of a CAG/CTG tandem repeat at a specific locus in the genome. In all cases, the longer the repeat tract, the more severe the disease phenotypes. Thus, an attractive hypothesis is that contracting the repeat tract to non-pathologic sizes will remove the underlying cause of the disease and thereby may slow, stop, or prevent disease progression. Here I present our work on developing a gene editing approach to contract expanded repeat tracts. It relies on a mutated Cas9 (D10A), which turns the enzyme into a nickase. Expressing the Cas9 nickase together with a guide RNA that targets it to the repeat tract itself efficiently contracts expanded repeats at multiple loci and in multiple cell types. The approach does not appear to induce a significant number of off-target mutations or on-target rearrangements and does not change the size of tracts within the non-pathogenic range. Together this approach may represent a novel therapeutic avenue worth translating into the clinic.
To view the video assoicated with this lecture click here
Youtube

Hosted By

ELRIG
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'
Login
Eventflo Home
copyright ELRIG, eventflo.co.uk, Labhoo Ltd 2003-2021