Research highlights and societal impact

Prion diseases such as Creutzfeldt-Jakob disease (CJD) are the most rapidly fatal dementias and are without effective treatments. They result from the transformation of a normal cellular protein, the prion protein into infectious "prions" that induce nerve cells to degenerate.

In studies published in Nature, Dr Hawke has shown that targeting normal prion protein with antibodies suppresses this transformation. However, being large molecules, antibodies do not cross the intact blood brain barrier in sufficient quantities to inhibit the transformation in the CNS and current experiments in the Hawke lab are designed to turn off the expression of normal prion protein using gene knockdown technology.

With funding from the University of Sydney Medical Foundation (Jessie and Isabel Alberti Program grant) and the NHMRC, long-term experiments are in progress assessing the effectiveness of gene therapy in animal models of CJD. Here viruses expressing RNA molecules that target for destruction the mRNA translating the prion protein are introduced into mice challenged with infectious CJD prions. Preliminary results of these experiments will be available in the later half of 2009. If successful, this technology should be readily translatable into therapy for human prion diseases.