Designing gene editing for stem cells

Designing novel CRISPR-Cas based corrective gene editing solutions for mutations in hematopoietic stem cells that result in bone marrow failure syndromes

Degree type


Closing date

27 March 2023



Citizenship requirement


About the research project

Inherited Bone marrow failure syndromes (BMFS) are a rare disease with often disastrous consequences due to the underlying defects in the cells in the bone marrow that are needed to generate the wide array of cells that make up the blood.

We are in a very exciting era in which we are seeing CRISPR-based gene editing products entering the clinic. For the first time it is possible to correct the underlying genetic mutation that is disease causative. We have been working with CRISPR editing for several years and have recently received MRFF funding to design and test CRISPR-based gene editing in hematopoietic stem cells. Should we be successful in editing hematopoietic stem cells these cells are able to regenerate the entire blood system of an individual, and would be curative. Notably small numbers of hematopoietic stem cells have been shown to be sufficient to regenerate the entire blood system. The advantage of this approach over what is current standard of care, a Bone Marrow Transplant (BMT), is that by using the patient’s own cells we will avoid graft versus host disease, the most common adverse event associated with BMT.

This project will involve designing and generating CRISPR gene editing products, and working with collaborators in Victoria, South Australia and New South Wales to develop mRNA-LNP delivery methods based to deliver these constructs to hematopoietic stem cells. HSCs will be tested in repopulation studies.

Primary Supervisor

Meet Dr Kirsten Fairfax


Applicants will be considered for a Research Training Program (RTP) scholarship or Tasmania Graduate Research Scholarship (TGRS) which, if successful, provides:

  • a living allowance stipend of $31,500 per annum (2023 rate, indexed annually) for 3.5 years
  • a relocation allowance of up to $2,000
  • a tuition fees offset covering the cost of tuition fees for up to four years (domestic applicants only)

If successful, international applicants will receive a University of Tasmania Fees Offset for up to four years.

As part of the application process you may indicate if you do not wish to be considered for scholarship funding.


Applicants should review the Higher Degree by Research minimum entry requirements.

Additional eligibility criteria specific to this project/scholarship:

  • Applications are open to Domestic or International applicants
  • Applications are open to applicants with experience in molecular biology
  • English language score must be above minimum entry requirements for this project
  • Applicants must be able to undertake the project on-campus

Selection Criteria

The project is competitively assessed and awarded.  Selection is based on academic merit and suitability to the project as determined by the College.

Additional desirable selection criteria specific to this project:

  • Previous experience with cell culture and culturing primary cells

Application process

There is a three-step application process:

  1. Select your project, and check you meet the eligibility and selection criteria;
  2. Contact the Primary Supervisor, Dr Kirsten Fairfax to discuss your suitability and the project's requirements; and
  3. Submit an application by the closing date listed above.
    • Copy and paste the title of the project from this advertisement into your application. If you don’t correctly do this your application may be rejected.
    • As part of your application, you will be required to submit a covering letter, a CV including 2 x referees and your project research proposal.

Following the application closing date applications will be assessed within the College. Applicants should expect to receive notification of the outcome by email by the advertised outcome date.

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