The exciting field of small RNA research was the subject of the 2006 Nobel Prize in Medicine, and holds great potential in the diagnosis and prognosis of disease such as cancer. Recent clinical studies suggest that drugs inhibiting small RNAs called microRNA present novel therapeutic opportunities. By defining the non-specific effects of such drugs and investigating new avenues for their delivery, this project will secure the safe application of these drugs into the clinic.
Identification And Evaluation Of Novel Epigenetic Targets For The Treatment Of ? Haemoglobin Disorders
Funder
National Health and Medical Research Council
Funding Amount
$740,809.00
Summary
Symptoms of ?-haemoglobin disorders appear after birth, when fetal haemoglobin (HbF) is replaced by aberrant adult haemoglobin (HbA). Interestingly, the persistent expression of HbF in patients reduces disease severity. This observation has created much interest in understanding the fetal to adult transition, since reversing it can benefit patients. Epigenetics plays a central role to this mechanism. Identifying components of this switch will form the basis of next generation therapies.
Development Of Novel Gene Therapy Vectors For Thalassaemia
Funder
National Health and Medical Research Council
Funding Amount
$287,307.00
Summary
Thalassaemia, is a common inherited disorder affecting haemoglobin synthesis. Synthesis of ?/?-globin chain is balanced during normal red blood cell production. Any disruption in the ratio of ?/?-globin chain results in anaemia. In this study, we will explore gene therapy strategies to restore balanced ?:? globin expression and ultimately improve the severely anaemic phenotype in ?-thalassaemia patients.
Thalassaemia, is a common inherited disorder affecting haemoglobin synthesis. Synthesis of ?/?-globin chain is balanced during normal red blood cell production. Any disruption in the ratio of ?/?-globin chain results in anaemia. In this study, we will explore gene therapy strategies to restore balanced ?:? globin expression and ultimately improve the severely anaemic phenotype in ?-thalassaemia patients.