CRISPR therapy development
This genome editing project focuses on developing CRISPR gene therapy for Duchenne Muscular Dystrophy (DMD).
The long term goal of this project is to develop innovative CRISPR gene therapy for Duchenne Muscular Dystrophy (DMD). DMD is a devastating muscle wasting disorder that affects 1:5,000 male births. Muscle weakness usually begins around the age of 4 and by age 12 most affected boys are in a wheelchair.
DMD is caused by mutations in the X-linked DMD gene, which encodes the muscle protein Dystrophin. The first aim of this project is to use CRISPR to generate and characterise a DMD mutant mouse that models a common disease-causing mutation in affected boys. Once the preclinical model is established, we will deliver optimised CRISPR reagents to the muscle tissue to edit the defective gene and restore its function.
If successful, we aim to translate our strategy into human patients. The approaches developed in this project could also potentially be applied to the development of CRISPR gene editing therapy for other inherited disorders.
Study genome editing
CRISPR genome editing technology is transforming medicine, biology and agriculture. CRISPR enables targeted genetic modification of virtually any species with unprecedented efficiency. Given its potential, CRISPR is envisioned to be a game changer for therapeutic development, particularly for incurable genetic diseases.
The Genome Editing Laboratory led by Professor Paul Thomas uses state-of-the-art molecular genetic approaches to develop CRISPR technology to enhance human health. Our CRISPR innovation includes the development of strategies to eliminate entire chromosomes that could potentially be deployed for treatment of aneuploidy diseases.
Our lab is expert in generating genetically modified mice using CRISPR to model human disease mutations with >60 mouse models to date. We are using these unique models to investigate the pathology of relatively common genetic diseases such as epilepsy and muscular dystrophy. We are also developing CRISPR genome editing approaches to cure genetic diseases by correcting disease-causing mutations in vivo.
Finally, we are leading the world in the development of CRISPR gene drives in mice. This powerful technology has enormous potential for controlling invasive mouse populations that spread (zoonotic) disease, cause species extinction and loss of agricultural productivity.