There are four basic objectives to the proposal:
1. Development of new constructs which express antiviral genes directed against HIV.
2. Demonstration of expression of these genes in eukaryotic cells. Stable and, where appropriate, inducible expression.
3. Assessment of the ability of these antiviral genes to inhibit replication of HIV which has been introduced into these cells.
4. Efficient delivery of such antiviral constructs, firstly to established the cell lines, secondly to primary human T-cells derived from peripheral blood, and thirdly to haemopoietic stem cells. These will be derived from CD34 cells from umbilical cord and bone marrow cell populations or from peripheral blood.
A necessary accompaniment to these studies is that the delivery of such antiviral constructs should be performed in as safe a manner as possible. Thus, ensuring safety will be paramount during these experiments and in the gene therapeutics being developed.
These objectives are similar to those currently being attempted by groups elsewhere, notably in the USA. Our grouping however has an unusual combination of expertise in the diverse areas of gene delivery and in HIV molecular biology. All the collaborators are experts within their fields. Whilst it is possible that commercially exploitable gene therapeutics will derive from this collaboration it is not certain there is an equivalent group in industry.