Unit Director: Prof Patrick Arbuthnot
Detailed information on gene structure and DNA sequences has enabled investigation of rational approaches to modifying genes as a means of treating disease. Since genes underlie almost all biological mechanisms, including pathological processes, the potential for controlled alteration of gene function is enormous.
The term ‘Gene Therapy’ was coined in the 1970s and refers to the use of procedures that are intended to alleviate disease by genetically modifying the cells of a patient. To restore the health of cells, gene therapy may involve repairing damaged genes and silencing ‘rogue’ genetic elements that may be found during viral infection. The focus of the SAMRC/Wits Antiviral Gene Therapy Research Unit (AGTRU) is on developing use of nucleic acids as therapies to inhibit replication of viruses that are particularly important to sub-Saharan Africa.
Earlier research in the unit has been concerned with harnessing the RNA interference (RNAi) pathway and using gene editing to inhibit replication of hepatitis B virus gene (HBV), HCV, HIV-1 and Rift Valley Fever viruses. Globally, these viruses are significant causes of public health problems and available therapies have modest curative efficacy. HBV infection, the main topic of research of the SAMRC/Wits AGTRU, is hyperendemic to sub Saharan Africa and carriers of the virus are at high risk for cirrhosis and liver cancer. The improvement of currently available HBV therapies takes top priority so as to prevent complicating cirrhosis and hepatocellular carcinoma. Having previously demonstrated successful inhibition of replication of antiviral gene-disabling technologies, emphasis has now shifted to the development of clinically relevant safe and efficient vectors (carriers) of the antiviral nucleic acids, and also the engineering of ‘designer’ sequence-specific DNA binding proteins.
Central to our goal as a unit is nurturing strategic South African and international collaborations with academic and industrial partners so as to translate the powerful technology to clinical application.
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