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A PhD scholarship is offered, for a student to work on the project: Development of a universal gene therapy approach using CRISPR-based genome editing technology to treat paediatric liver disease.
This PhD project aims to develop a universal genome editing platform technology that can be easily adapted to treat urea-cycle disorders and liver disease more broadly. This approach has the advantage of correcting all mutations within the target gene regardless of their type and could, therefore, treat all patients with the same gene therapy vector, ensuring the greatest clinical applicability. In addition, the reagents generated in this project will be configured with the end-in-mind and will be directly translatable without further modification. The project will also identify novel, highly human liver-specific, vectors with the potential to generate intellectual property and commercial interest. Studies will be performed in patient-specific primary human hepatocytes in vivo, the most relevant pre-clinical model for human translation.
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.
Benefits
This scholarship is valued at $40,000 per annum and is tenable for up to 3.5 years.
Who's eligible
You must:
Preference will be given to applicants who can demonstrate they have experience in one or more of the following:
Background
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
Applications MUST be submitted here: https://www.sydney.edu.au/scholarships/c/scholarship-in-the-development-of-a-universal-gene-therapy-appro.html
Closing date: 11 February 2022 at 11.59 pm
Contact Dr Samantha Ginn for more information: sginn@cmri.org.au