Induction Of Antigen-specific Humoral Tolerance By RAAV-mediated Delivery Of CTLA4-Ig-antigen Fusion Molecules
Funder
National Health and Medical Research Council
Funding Amount
$524,456.00
Summary
There are many medical situations where immune suppression is required. Available methods lack specificity and risk infection, drug-related side-effects and cancer. We have discovered a novel way of suppressing immunity such that only unwanted responses are eliminated. This involves virus-mediated delivery of antigen fused to CTLA4-Ig. We plan to test this strategy in the context of gene therapy, to work out how it works and to optimise the approach. Success will have broad health implications.
Development Of Lentiviral Vectors For The Treatment Of X-linked Severe Combined Immunodeficiency (SCID-X1)
Funder
National Health and Medical Research Council
Funding Amount
$71,434.00
Summary
The first successful gene therapy clinical trial was reported in 2000 with the treatment of X-linked severe combined immunodeficiency (SCID-X1), commonly known as “bubble-boy” disease. The subsequent development of leukaemia in 3 of 11 patients has prompted the need to develop alternative vectors for gene delivery, such as HIV-1-based lentiviral vectors. This project will evaluate the efficacy and safety of lentiviral vectors in vivo, and hence their therapeutic potential for treating SCID-X1.