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

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/477112

Funding Status
Closed

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Funded Activity 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.

Funded Activity Details

Start Date: 01-01-2008

End Date: 01-01-2011

Funding Scheme: NHMRC Postgraduate Scholarships

Funding Amount: $71,434.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

Gene Therapy

ANZSRC Socio-Economic Objective (SEO)

There are no SEO codes available for this funding activity

Other Keywords

Adeno-associated virus | Gene Delivery | Gene Therapy | Genetic Disease | Liver

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