Finding cures for children's genetic diseases


For Researchers


The partnership between CMRI and CHW provides an excellent environment to undertake the challenging task of translating progress in the laboratory to new therapies in the clinic. While rewarding, this translational journey is immensely challenging as it demands effort across many fronts. In addition to laboratory research activities, we have developed specialised clean-room facilities for the genetic repair of patient cells, undertaken production of clinical grade gene delivery formulations, and formulated standardised procedures governing all aspects of clinical trial activity, all in compliance with complex regulatory requirements.

Research Areas

Clinical trials

We currently have SCID-X1 gene therapy trials underway, as well as trials aimed at treating late-stage brain tumours.

Other laboratory studies

In addition to our work on gene therapy for diseases of the bone marrow and liver, we are also exploring complimentary cell-based therapies for liver disease. Using genetic strategies, it is now possible to reprogram readily accessible cell types, such as skin cells, into cells of potential therapeutic importance, such as liver cells.

We have successfully reprogrammed mouse skin cells and are currently developing genetic repair strategies and defining the conditions that direct cells to become liver cells. Professor Tam’s group, with whom we are collaborating on this project, is particularly important in this regard, as knowledge of how cells grow and adopt specific functions during embryonic development provides important clues as to how to reprogram cells for therapeutic purposes.