Hurler syndrome is a single gene disorder causing the deficiency of an important protein found throughout the body. Affected children suffer mental deterioration, have problems with many of their internal organs and rarely live beyond 10 years of age. Bone marrow transplantation is the current therapy but is limited by the availability of donors and the frequent rejection of the transplanted cells.

Through genetic manipulation of the patient’s own bone marrow stem cells, the protein deficiency can be corrected. Using particular stem cells which have the potential to develop into many different types of tissue, Dr Grainger will investigate improved therapy for Hurler syndrome.