Development of lentiviral vectors for the treatment of X-linked severe combined immunodeficiency (SCID-X1) [ 2008 - 2011 ]

Research Grant

Researchers: Dr Claire Deakin (Principal investigator)

Brief description 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.

Funding Amount $AUD 71,434.88

Funding Scheme NHMRC Postgraduate Scholarships

Notes Dora Lush Biomedical Postgraduate Scholarship