Dr Marguerite Evans-Galea
Murdoch Childrens Research Institute
Rotary Club of Brighton North
Friedreich Ataxia, 2009
Dr Marguerite Evans-Galea is a Senior Research Officer with the Bruce Lefroy Centre for Genetic Health Research at the Murdoch Childrens Research Institute (MCRI). Dr Evans-Galea completed her PhD in 1999 at the University of New South Wales where she gained rigorous training in genetics and molecular biology and developed a strong understanding of the oxidative stress response.
From 1999 to 2007, Dr Evans-Galea gained extensive postdoctoral experience in the USA. At the University of Utah she characterised proteins involved in iron and zinc homeostasis. In 2001 she accepted a fellowship at St Jude Children's Research Hospital in Memphis, Tennessee. She made a key contribution by improving the safety of vectors for gene therapy.
In 2008, Dr Evans-Galea returned to her native Australia to pursue her scientific career. She has initiated two novel projects in the Bruce Lefroy Centre at the MCRI. Her research focuses on the development of potential therapies for the neurological disorder Friedreich ataxia.
Dr Evans-Galea has actively served as office bearer and member on numerous education and advisory committees, and several associations throughout her career. She has also enjoyed lecturing, supervising and training postdoctoral fellows, students and staff, both in Australia and the USA. Dr Evans-Galea is a strong advocate for biomedical research, mentoring and career development through a variety of media and events. Her broad training crosses multiple disciplines and model systems, and her experience in gene therapy complements her previous training to best equip her to transition this research from bench to the bedside.
SUMMARY OF PROJECT:
Lentiviral Gene Therapy for Friedreich Ataxia
Friedreich ataxia (FRDA) is an inherited neurodegenerative disease that affects 1 in 29,000 people. FRDA causes progressive damage to the nervous system and wide ranging neurological symptoms including unsteadiness that ultimately results in affected individuals requiring a wheelchair. A shortened life span is characteristic of FRDA with most individuals diagnosed before the age of 20.
FRDA is caused by an expansion in the frataxin gene which leads to reduced levels of frataxin, a protein important in intracellular iron metabolism. Debilitating diseases such as FRDA have limited options for treatment. Current therapies aim to improve patients’ quality of life but none can slow the progression of neurodegeneration, let alone cure the disease. This research project aims to develop gene therapy for FRDA by delivering unaffected copies of the damaged gene to cells.
Dr Evans-Galea’s studies will provide the foundation for the development of new therapies, specifically gene therapy, for people with FRDA.