- $140,000 scholarship for a PhD student in the Faculty of Medicine and Health (University of Sydney) to undertake research into AAV-Mediated Gene Transfer Vectors.
- Work with one of Australia’s most respected national and independent medical research institutes
- Located in Westmead, one of Sydney’s and Australia’s major biomedical research hubs
A PhD scholarship is offered, for a student to work on the project: Extending the therapeutic reach and biosafety of AAV-mediated gene transfer vectors to treat human metabolic liver disease: In vivo reactivation of the ornithine transcarbamylase locus in patient cells with skewed X-chromosome inactivation.
This PhD project’s primary objective, developing novel AAV-derived vectors with extended clinical reach for human liver disorders, will be translationally focussed on treating a frontier liver indication. Whilst exciting, treatment of haemophilias by AAV-mediated gene therapy succeeded because gene transfer to a modest proportion of hepatocytes confers therapeutic benefit. The gene transfer efficiency required to treat cell-autonomous indications is far higher, and rAAV technology is presently constrained by the low liver transduction efficiency of clinically deployed capsids. Ornithine transcarbamylase (OTC) deficiency is one such condition that demands the highest-efficiency liver-targeting technology for a clinically corrective intervention. Accordingly, we aim to employ our novel vectors to deliver locus-specific epigenetic engineering payloads to reactivate the wildtype OTC allele in hepatocytes.
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. CMRI is located at Westmead, a major hub for research and medicine in NSW, and is affiliated with the University of Sydney. We are easy to access by public transport.
This scholarship is valued at $40,000 per annum and is tenable for up to 3.5 years.
- be an Australian Citizen, Australian Permanent Resident or hold an appropriate visa that provides residency status and the right to work in Australia until the later of the 30 August 2023 or the projected conclusion of your PhD candidature
- be willing to apply for admission, applied for admission, have a conditional or unconditional offer of admission to commence or be currently enrolled in a full-time PhD within the Faculty of Medicine and Health
- conduct research as part of a project named, ‘Extending the therapeutic reach and biosafety of AAV-mediated gene transfer vectors to treat human metabolic liver disease: In vivo reactivation of the ornithine transcarbamylase locus in patient cells with skewed X-chromosome inactivation.'
- be able to demonstrate you hold an undergraduate degree with at least a credit average or equivalent and have either:
- research experience through an honours program (First Class or Second Class, Division I), or
- a Masters Degree by Research in a relevant subject.
Preference will be given to applicants who can demonstrate they have experience in one or more of the following:
- molecular cloning
- tissue culture
- animal handling
- vector production, and/or
- immunofluorescence and immunohistochemistry
This scholarship is funded by New South Wales (NSW) Health and the University of Sydney as part of the NSW Health Gene and Cell Therapy PhD Program.
Applications MUST be submitted here: https://www.sydney.edu.au/scholarships/c/therapeutic-reach-and-biosafety-of-aav-mediated-gene-transfer-ve.html
Closing date: 11 February 2022 at 11.59 pm
Contact Dr Samantha Ginn for more information: email@example.com