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

点击翻译按钮获取中文摘要

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.