Stem Cell Medicine | Murdoch Children’s Research Institute

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Nov 30 2018

Stem Cell Medicine is the Murdoch Children's Research Institute's (MCRI)world-class research program in stem cells.We havea vision to prepare Australia for the transition from fundamental stem cell research in the lab towardtranslation outcomes using stem cells, with the delivery of human stem cell-based products and clinical practices.

Our research includes stem cell-based disease modelling and drug screening and the development of stem cell therapies and bioengineered tissues (explained in detail below). Embedded in the Royal Children's Hospital, and located in Melbourne's biomedical precinct, MCRI Stem Cell Medicine ensures that we workclosely and collaboratively with key experts in the field, including clinicians, biomedical scientists, engineers, ethicists, as well asthe local and international biotech sector. We also engage closely with key stem cell research networks and bodies including Stem Cells Australia, the Australasian Society for Stem Cell Research, the National Stem Cell Foundation of Australia, and look forward to participating in the International Society for Stem Cell Research annual meeting scheduled in Melbourne 2018.

By consolidating our research talent, fostering collaboration, strategically investing resources, and expanding our infrastructureplatforms, MCRI is now one of the worlds leading institutes in pluripotent stem cell research.

A stem cell is a cell that can generate more stem cells but can also form other specialised types of cells.

The early embryo is formed from embryonic stem cells. These stem cells are pluripotent, which means they are able to divide and program themselves into any other type of cell. Pluripotent cells are no longer present after birth, and while some specialised adult stem cells remain throughout the body in places like the skin, liver, blood and intestine, most adult cells are fully differentiated.

In the lab, we reprogram these adult cell types back into pluripotent stem cells and then differentiate them again into the type of cell we wish to study. These are called induced pluripotent stem cells (iPSCs).

At MCRI, we turn iPSCs into different types of committed cells that the human body is unable to regenerate itself, such as the cells of the kidney and heart. Our researchers are world leaders in both generating iPSCs and generating specific cell types from them.

Using Stem Cell Medicine to Transform Clinical Care and Patient Outcomes

Our vision is to be an Australian and international leader in navigating the transition forthe use of human iPSCs from the lab towardclinical use and practice.The use of iPSCs enables cutting-edgeopportunities to conductpatient-specific disease modelling, personalised drug screening, cell therapy and bioengineered organs built from stem cells.

Our location within the Royal Childrens Hospital and our participation in theMelbourne ChildrensCampusmakes us ideally placed to discover and deliver stem cell medical breakthroughs.

Stem Cell Medicine Expertise

MCRI Stem Cell Medicinehas established a Stem Cell Derivation Facility for generating patient stem cells, and has pioneered protocols for turning these stem cells into heart, blood, cartilage, pancreas, nerves and kidney cells. In early 2017 we also introduced a Gene Editing Facility, currently servicing internally only.

We also have expertise in gene editing, which allows us to correct or create specific gene changes in normal or patient stem cells. Our Translational Genomics Unit is being used to examine tissues made from patient stem cells to monitor the effect of the gene changes on every other gene. This information can be used to validate the link between novel gene changes and disease, and find potential pathways to target with drugs.

Our expertise lies in the generation of stem cells from patients and the differentiation of those stem cells into different specialised tissues. This provides the potential for both patient-based disease modelling and the development of treatments for:

It is now possible to take any cell from a patient and turn this back into a stem cell, which we then have the ability to transform into any other cell type in our labs. This means we can make a stem cell from a patient with a disease and study any gene changes that may have caused the disease. This stem cell can then be differentiated into the cell type that is damaged, such as a nerve, kidney or muscle cell for a 'disease in a dish' approach.

In this way, we can study whether a gene is at fault, and then understand why this change is affecting cell function. Put simply, we make a tissue from the patient to understand their disease.

This provides the possibility of testing new drugs or new therapies to specifically treat that patient.

Human tissue formed using stem cells provides a new approach for the pharmaceutical industry to test drugs before clinical trial, providing an early opportunity to identify drug toxicity and a platform for testing drug efficacy.

Such drug screening has the potential to reduce the use of animals in drug development and enable substantial cost savings in the pharmaceutical industry. Using patient-derived stem cells, it may also be possible to develop personalised treatments by testing these on patient stem cell-derived tissues.

By improving our methods for turning a human stem cell into the many types of tissues present in the body, it will eventually be possible to deliver cells back into patients to treat disease.

Around the world, the first clinical trials using stem cell-derived tissues are being performed to treat blindness, neurological disease and diabetes.

Together with new approaches for making the right cell type, MCRI Stem Cell Medicine is ideally located within a hospital precinct, allowing research into how to deliver the right cells into the right patients and access to the Melbourne Childrens Trial Centre to test safety and patient outcome.

Our research will focus on the treatment of childhood cancer, blood disorders, heart disease and kidney disease.

In some cases, delivering a cell may not be enough. MCRI Stem Cell Medicine also conducts research into approaches for the bioengineering of replacement organs using human cell types generated from stem cells.

The hope is that this will ultimately provide treatments for many conditions, including heart disease, bone disease and kidney disease.

Delivering a cell or bioengineered organ into a patient will face the same challenges seen in conventional organ donation.

Without a good DNA match, the patient will reject their stem cell treatment. To address this, we aim to generate a bank of human stem cells matched to the genetically diverse Australian population. These stem cells, generated from banked cord blood, can be turned into the required cell type for treatment ensuring a good match for the patient being treated.

As the home of the Bone Marrow Donor Institute (BMDI) Cord Blood Bank, MCRI is ideally positioned to establish the first bank of human stem cells (known as a Haplobank) matched for the Australian population.

Tailored to the ethnic diversity present in Australia, we will generate human stem cell lines designed to provide a transplantation match for more than 95 per cent of the Australian population.

Stem cell research has attracted considerable public interest and ethical debate. We engage actively with the public to discuss this new area of research, listening to the feedback from the community on any potential concerns and ensuring there is accurate information available for those seeking advice.

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Project Manager Stem Cells

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Program Director Office Research

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Stem Cell Medicine | Murdoch Children's Research Institute

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