SUPPORTING INDIVIDUALS, FAMILIES AND RESEARCHERS
IN THE CYSTINOSIS COMMUNITY

Sunderland University Receive Funding From USA

Researchers at Sunderland University have recently received an £80,000 grant from the Cystinosis Research Network in the USA to help fund a new 3 year study that will focus on proteomic research (the study of proteins within cells).

This is exciting news for the team which has been involved in the field of Cystinosis research for some years under the leadership of Professor Roz Anderson. The team has already been supported for several years by the Cystinosis Foundation UK and this has led to successful research into the development of prodrugs – medicines that are designed to be targeted efficiently to the appropriate areas of the body which improves their absorption and effectiveness.

This new project complements the pro-drug research. Professor Anderson picks up the story…

Proteomic investigation of cystinotic cells and the effects of cysteamine treatment – A CRN funded project

Proteomics involves the study of the structure and function of proteins in biological systems; its application has allowed the characterisation of many diseases by their production of aberrant proteins in response to altered DNA and / or malfunctioning cellular biochemistry. This increase in protein information has enabled the identification of specific disease biomarkers and provided new opportunities for the treatment of particular diseases.

In this project, we aim to use proteomics to gain further knowledge about cystinotic cells in comparison to normal cells and the effects of treatment with cysteamine upon cystinotics cells. Through comparison of the proteins found in normal cells and cystinotic cells, we expect to identify differences in the cellular proteins, which may provide further insight into the disease. Further work comparing untreated and treated cystinotic cells
will investigate the changes caused by cysteamine to the cellular proteins; this has the potential to offer new information about the biochemistry of cystinotic cells and a better understanding of the beneficial and possible detrimental effects of cysteamine. We can use these techniques and data to ensure that the prodrugs result in the same beneficial effects as cysteamine and that they do not cause any adverse effects to the cells.

Another possible result would be the identification of biomarker proteins that can be used to monitor disease and treatment progression or to the recognition of other, possibly new, therapeutic intervention strategies.

Professor Roz Anderson, Sunderland University

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