Gene therapy to treat inherited brain disease

Biology, biomedical science and biotechnology

Gene therapy has held promise for treating single gene disorders by replacing the defective gene with a functional one.

Our laboratory is involved in clinical trials for inherited disorders that affect the brain.

One of the challenges for any treatment, including gene therapy, for these diseases is their progressive nature which means the residual disease burden already established prior to diagnosis of the disease is largely impermeable to therapeutic intervention.

Early treatment is key to improving outcome, and so is understanding the pathology of the neurological decline, which is relentless manifesting a loss of acquired skills e.g. the ability to speak, walk and eat.

In addition to the clinical samples, projects are also available using cell and mouse models of disease which are currently being used to undertake parallel studies to learn more about the progression of neurological damage, open up new avenues for treatment and adjunct therapies to complement gene therapy.

Researcher photo Maria Fuller


Professor Maria Fuller

Co-supervisorsDr Jennifer SavilleDr Nathan Rout-PittDr Ainslie Derrick-Roberts

Research area: Genetics and molecular pathology

Recommended honours enrolment: Honours in Molecular and Biomedical Science


Tagged in Honours projects - Molecular and biomedical science, Honours projects - Molecular and biomedical science: Genetics, Honours projects - Molecular and biomedical science: Biochemistry, Honours projects - Maria Fuller