Reconstitution of B-cell ontogeny in Btk-deficient patient-derived CD34+ cells in a humanised mouse model: The foundations for an Australian XLA gene therapy trial

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/1065036

Funding Status
Closed

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Funded Activity Summary

Gene therapy targeting the bone marrow has broad therapeutic potential in the management of genetic diseases, viral infections and cancers involving blood cells. In this proposal we plan to obtain bone marrow cells from patients with X-linked agammaglobulinaemia and show that their cells can be genetically repaired in the test tube and, when transferred back into a specialised mouse models, reconstitute the immune system. The results have the potential to underpin a human clinical trial.

Funded Activity Details

Start Date: 01-01-2014

End Date: 01-01-2016

Funding Scheme: Project Grants

Funding Amount: $678,652.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

Genetics not elsewhere classified

ANZSRC Socio-Economic Objective (SEO)

There are no SEO codes available for this funding activity

Other Keywords

bone marrow | child health | gene delivery | gene therapy | virus

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