Table of Contents

HK J Paediatr (New Series)
Vol 5. No. 1, 2000

HK J Paediatr (New Series) 2000;5:88

Proceedings of 2nd Hong Kong Medical Genetics Conference

Gene Therapy

HYS Ngan

HK J Paediatr (new series) 2000;5:85-89

Proceedings of 2nd Hong Kong Medical Genetics Conference, Hong Kong Society of Medical Genetics (Selected Abstracts)
15-17 October, 1999

Gene therapy is the introduction of functioning genes into a cell to correct an inborn error or dysfunctional gene or to add new function to a cell. The addition of gene has the potential to cure monogenic defect such as hemophilia B, familial hyper-cholesterolemia. Trials are carried out in potentially lethal genetic defective disease. Less well defined genetic diseases such as diabetes mellitus, cancers may also benefited from gene therapy. In cancer treatment, gene therapy not only confined to gene replacement, but involves strategies such as immunotherapy, chemotherapy sensitization and anti-oncogenic therapy.

Techniques consisted of first cloning the gene, select cell target and gene transfer. Gene can be transferred by physiochemical methods such as liposomes, naked plasmid DNA and gene gun or viral vectors such as adenovirus and retrovirus.

Potential problems of gene therapy include choice of gene to be transferred, how to enhance the delivery of the gene (transfer efficiency) into targeted cells (transfer specificity), how to control the expression of inserted gene, how to lower the immune response of host to vectors if viral vector is used.

Our in-vitro study using p53 expressing adenovirus transfer to cervical cancer cell lines showed inhibition of growth as well as apoptosis. Clinical trials in other centres showed encouraging results in advanced cancer treatment such as in carcinoma of lung. To conclude, though there is a potential for gene therapy, it is not ready for clinical practise yet.


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