Targeted Gene Therapy for Hepatocellular Carcinoma

 viral hepatitis

Dr Marco Della Peruta, postdoctoral scientist, at work in the lab.  To hear Marco talking about his work please see our short film at:


One of the most remarkable features of the liver is its ability to self-repair by producing replacement liver cells, or hepatocytes. In patients with Hepatocellular Carcinoma (HCC), the liver's ability to produce these healthy hepatocytes becomes impaired and cancerous cells begin to proliferate. 

Our research staff working in this area plan to exploit recent insights into the development, or pathogenesis, of  (HCC) in conjunction with gene transfer technology to deliver a therapeutic gene directly to the liver to target and destroy cancerous cells. This 'magic bullet' will be carried to the liver by a viral vector, administered to the patient by a single injection. Research to date has shown that vectors based on adeno-assocviated viral vectors (rAAV) are best suited for gene transfer approaches in the treatment of HCC.


The specific aims of this Group are:-

1.  To develop an expression cassette that limits expression of a therapeutic transgene in HCC cells in preference to normal hepatocytes, thus limiting toxicity.

2.  To determine if the addition of alpha fetoprotein enhancer/promoter elements to the AAV expression cassette further restricts expression of the transgenes to tumour cells.

3.  To use the optimised expression cassette to mediate selective transgene expression in HCC and not in the surrounding normal tissues


The work is now at the stage where pre-clinical models have shown very promising results and the aim is to move into trials with patients within the next 12 months.


Published by: Foundation for Liver Research

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