Correction Of DMD Mutations Using Genome Surgery

What Is Genome Surgery?

Professor George Dickson works at the Royal Holloway University, London. His team is working on a technique called genome surgery, in which the genetic mutations that cause DMD can be removed. This technique is achieved by using enzymes called endonucleases, when injected into cells these act like molecular scissors and cut out the DNA mutation. The next step is to add in the correct DNA sequence and then stitch the cut ends together.

The molecular tools required for the gene surgery technique were generated and then tested in cell culture models of Duchenne muscular dystrophy and in a mouse model.

Genonome surgery is a techque which could treat all those with Duchenne muscular dystrophy whatever their mutation

What Is Happening About Clinical Trials?

This project is in the preclinical stage which means it is not yet ready to be tested on humans.

The project was funded through the Duchenne Forum. The other members of the Duchenne Forum are MDUK, The Duchenne Research Fund, Harrison's Fund and Alex's Wish.

Published on 4 May 2016

Share this article