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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.

Professor Paul Thomas

Supervisors

Professor Paul Thomas

Co-supervisor: Dr Fatwa Adikusuma

Research area: Genome editing

Recommended honours enrolment: Honours in Molecular and Biomedical Science

Tagged in Honours projects - Molecular and biomedical science, Honours projects - Paul Thomas, Honours projects - Fatwa Adikusuma, Honours projects - Molecular and biomedical science: Biochemistry