Molecular Medicine

Molecular Medicine

Green staining of microtubules
Researchers in the Molecular Medicine division are investigating how biological systems function and are controlled in normal and disease states.
With programs focused on blood cell production and function, epigenetics and cancer, our goal is to pinpoint molecular targets for disease diagnosis and treatment.

New therapies for muscular dystrophy

Facioscapulohumeral muscular dystrophy (FSHD) is a progressive wasting disease that aff ects the face, arms and shoulders. No treatments are currently available.

Associate Professor Marnie Blewitt and colleagues are investigating the gene Smchd1, which plays a role in switching other genes off. In FSHD, genetic changes in SMCHD1 prevent it from working properly, contributing to disease. By understanding the function of Smchd1, the team hopes in the long-term to develop drugs that help people with FSHD.

Understanding platelet production

Platelets are tiny blood cells essential for clotting. Learning how platelets are produced is key to understanding diseases where too many or too few platelets are made.

Dr Samir Taoudi and colleagues are comparing the genes that control platelet development before birth versus in the adult.

The team discovered prenatal platelet-forming cells do not require the Mpl gene, which is an essential regulator of platelet development in the adult. This indicates there are different processes contributing to prenatal and adult platelet development.

A fine-grained look at biology

Cell populations, tissues and organisms have traditionally been studied in bulk, masking processes that occur in individual cells.

Research in the Molecular Medicine division has used recent technological developments that are spearheading an appreciation of biology at the single cell level. Single cell RNA sequencing provides insight into which genes are switched on within an individual cell. A genetic technique called ‘cellular barcoding’ traces behaviour of single cells within a population.

These techniques, along with novel computational tools, are unmasking cell types that are hidden in conventional analyses of cells at the population level.

Health impact

Cancers: blood cancers, leukaemia

Immune disorders: allergy, asthma, multiple sclerosis, rheumatoid arthritis

Other areas: developmental biology, epigenetics, muscular dystrophy, personalised medicine, regenerative medicine

Division head

Professor Doug Hilton

Laboratory heads

Associate Professor Marnie Blewitt

Dr Shalin Naik (jointly with Immunology division)

Dr Matthew Ritchie

Dr Samir Taoudi (jointly with Cancer and Haematology division)

Division coordinators

Sonja Gustin

Fiona McGrath