Research Supervisor Connect
OI or osteogenesis imperfecta is a congenital bone fragility disorder that results in frequent fractures and a tremendous impact on quality of life. In individuals with severe OI, the condition can be deforming and restrict them to wheelchairs. Our group has developed adeno-associated viral (AAV) vectors able to target bone and are combining them with CRISPR-Cas9 gene editing technology to treat OI. This PhD project will focus on cell culture and preclinical models of bone disease as well as emerging gene therapy approaches. The research findings will have the potential to transform the treatment of patients with OI.
Associate Professor Aaron Schindeler.
Westmead - Westmead Institute for Medical Research
This project involves the development and/or use of cell lines gene modified to contain OI patient mutations. Mutations are introduced using CRISPR-HDR. Models of type IV and type V OI are currently being prioritized. Gene rescue studies will be performed in cell lines to maximize editing efficiency and avoid off-target effects. Promising therapies will be next applied to preclinical mouse models with delivery using AAV vectors. Outcome measures will be both genetic and functional, and our laboratory has access to state-of-the-art bone-imaging equipment.
We are seeking motivated students interested in developing their technical skills and wishing to make a difference in the lives of children with severe genetic disorders. This project is ideal as a PhD project, but a modified version of a Masters project could be a possibility. A strong candidate will be able to work as part of a multidisciplinary research team and have strong written/oral communication skills. The capacity to attract an independent scholarship (first class honours, prior publications) will be looked upon extremely favourably.
Co-supervisor Samantha Ginn sginn@cmri.org.au
For more information contact A/Prof Aaron Schindeler at aaron.schindeler@sydney.edu.au
The opportunity ID for this research opportunity is 3415