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Gene therapy for genetic and acquired diseases of the liver and haematopoietic system.


We are working on developing new therapies for the treatment of genetic and acquired diseases of the liver and haematopoietic system.

The Gene Therapy Research Unit has a diverse selection of projects available that can be tailored for honours or PhD student. All projects focus on the development and use of gene transfer and genome editing technology.


Professor Ian Alexander, Dr Samantha Ginn, Dr Grant Logan.

Research location

Westmead - Childrens Medical Research Institute

Program type



The liver and haematopoietic system are "holy grail" targets for gene therapy. Mastery of the challenges of gene transfer and genome editing in these organ systems opens the prospect of correcting a host of clinically troubling disease phenotypes. Achieving stable genetic repair in a sufficient number of cells to confer clinical benefit remains a major challenge. Projects available within the Gene Therapy Research Unit focus on further developing gene transfer and genome editing technology, strategies for selectively expanding genetically repaired cells and the application of these technologies to the treatment of mouse models of human disease.

Available project areas include:
1) Isolation and vectorization of novel adeno-associated viruses from Australian fauna
2) Development of genome editing technologies and strategies in hepatocytes and bone marrow stem cells
3) Safety of adeno-associated virus vectors in the human liver
4) Engineering of adeno-associated virus capsid proteins for enhanced tropism in human cells
5) Selective expansion of gene-modified hepatocytes in vivo
6) Development of strategies for stable gene transfer to the growing liver
7) Disease modeling and therapy in the chimeric mouse-human liver

Additional information

Cell culture
Recombinant DNA technology
Recombinant virus development, construction and production
Gene transfer and genome editing in vitro and in vivo
Modeling human disease and therapy in small animal models

PhD entry: Hons I classification, lab-based research experience is preferable. Children’s Medical Research Institute (CMRI) is an award-winning state-of-the-art medical research facility, with over 100 full-time scientists dedicated to researching the genes and proteins important for health and human development. The CMRI is supported in part by its key fundraiser Jeans for Genes®. Our scientists are internationally recognised research leaders and foster excellence in postgraduate training. CMRI graduates are highly sought after nationally and internationally.  

CMRI is located at Westmead, a major hub for research and medicine in NSW, and is affiliated with the University of Sydney. Easy to access by public transport.  

We are looking for top quality students who can prove a dedicated interest and enthusiasm for scientific research. 

Candidates may apply for a CMRI PhD Research Award, which exceeds the Australian Postgraduate Awards and NHMRC scholarships in value. Visit the CMRI website for more details.

Want to find out more?

Opportunity ID

The opportunity ID for this research opportunity is 2102