New therapies for childhood dementia

A new substrate reduction strategy to treat multiple childhood dementias

Degree type


Closing date

27 March 2023



Citizenship requirement


About the research project

There are more than 70 rare genetic diseases that cause childhood dementia, which affect the child’s ability to learn, play and recognise family and friends. Most of these result in childhood death and do not have disease-modifying treatment options. The burden of childhood dementia is largely hidden, but is similar in prevalence to motor neurone disease, has an incidence comparable to cystic fibrosis, and has a death rate on par with childhood cancer. Lysosomal storage disorders (LSDs) are the cause of most childhood dementias, resulting from homozygous or compound heterozygous loss-of-function mutation of genes encoding lysosomal enzymes. These enzymes catalyse reactions that breakdown macromolecules, but in lysosomal storage disorders, this enzyme deficiency results in accumulation (‘storage’) of such molecules. One strategy for treating LSDs is to reduce the amount of macromolecules being generated (known as substrate reduction therapy). Many LSDs causing childhood dementia result in accumulation of glycosphingolipids within lysosomes, however there are currently few treatment options. In these diseases, glycosphingolipid accumulation is the primary pathological feature that results from lysosomal enzyme deficiency, or are secondary pathological features caused by disruption of other lysosomal functions.

This project will develop and test new approaches to reducing accumulation of glycosphingolipids in disease-specific models of childhood dementia. You will work as part of a team that includes basic scientists, clinicians, and patient advocacy groups, and gain broad expertise in contemporary techniques in cell and molecular biology, including induced pluripotent stem cell generation and differentiation, 'omics technologies, and electrophysiology.

Primary Supervisor

Meet AProf Tony Cook


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 applications from neuroscience/cell biology/molecular biology disciplines or similar background only
  • 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 essential selection criteria specific to this project:

  • Strong communication skills
  • Strong record keeping skills
  • Familiar with PC2 laboratory research

Additional desirable selection criteria specific to this project:

  • Experience in contemporary cell-based techniques, particularly iPSC culture and differentiation, and/or CRISPR/Cas gene editing
  • Experience in image-based analyses

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, AProf Tony Cook 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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