Explorer Grant targets cure for life-shortening disorders

New Explorer Grant targets cure for life-shortening genetic disorders

One of the recipients of the Health Research Council of New Zealand’s (HRC) new Explorer Grants will use his $150,000 award to test a new technology that could potentially replace the defective genes responsible for such incurable genetic disorders as cystic fibrosis.

University of Auckland researcher Associate Professor Geoffrey Krissansen will use his Explorer Grant from the HRC to examine whether a new protein replacement therapy technology can combat cystic fibrosis and other genetic disorders like hereditary diffuse gastric cancer (a specific type of stomach cancer), both of which are caused by a defect to a single gene.

A new drug delivery agent developed by Dr Krissansen and his colleagues will be used to deliver drugs directly to damaged epithelial cells that line the skin, respiratory and gastrointestinal tracts.

“There are more than 6,000 known single-gene disorders, which occur in about 1 out of every 200 births. Congenital mutations in single genes result in devastating diseases of the skin and other epithelia,” says Dr Krissansen.

“We propose to employ novel drug delivery technology to replace the defective proteins associated with each disease to provide a potential cure. The technology has the potential to transform medical practice in the prevention or treatment of genetic disorders that are currently incurable,” he says.

“This is the first year that the HRC has awarded Explorer Grants to health researchers,” says HRC Chief Executive, Dr Robin Olds. “The grants – three in total – are designed to provide seed support for projects that advance transformative, innovative, exploratory or unconventional ideas, and which have the potential for major impact.”

Genes – this time those contained in the mitochondria or “powerhouses” of cells – are also the focus of Professor Michael Berridge’s Explorer Grant project. Professor Berridge from the Malaghan Institute of Medical Research in Wellington will use his grant to develop technology to manipulate mitochondrial DNA in cells.

At least 200 human diseases are reported to be due to mitochondrial gene mutations.

“The inability to alter mitochondrial genomes1 in cells has hindered research into the many diseases caused or contributed to by mitochondrial mutations. Our proposal seeks to directly address this challenge and transform the field of mitochondrial biology by developing technology to replace natural mitochondrial genomes in cells with custom-designed synthetic genomes,” says Professor Berridge.

Dr Michael Eccles from the University of Otago, Dunedin, has been awarded an Explorer Grant to investigate a new way to measure the aggressiveness of cancer cells using circular polarised light.

“Our new optical methodology measures increased aggressiveness in cancer cells due to changes that occur in the optical properties of the cells. These changes result from the increasing nuclear to cytoplasm ratio as the cancer cells become progressively less differentiated2 and more aggressive,” explains Dr Eccles.

The grant will help Dr Eccles and his colleagues determine whether this method can be used to identify and grade live cancer cells in situ. If successful, the project has the potential to revolutionise techniques used for cancer cell detection and cancer diagnosis.
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