A specialist in genetic medicine at the National Institute for Health Research’s Manchester Biomedical Research Centre is leading a multi-national team investigating the genetics of immune system disorders.
Professor Yanick Crow has been awarded a European Union grant of €5.4 million over three years to investigate Nuclease Immune Mediated Brain and Lupus-like (NIMBL) conditions. These are devastating genetic disorders which lead to greatly reduced quality of life, high mortality especially in children, and significant risks of recurrence within affected families. NIMBL conditions are rare, but under-diagnosed. No effective treatments or cures currently exist.
“To help us provide the best care for patients worldwide, we need a better understanding of the natural course of these disorders and why they occur,” explained Professor Crow, who is co-ordinating a team of researchers from Italy, the Netherlands, Spain, the UK and the USA.
“Through the NIMBL project, we will collaborate to develop a shared approach to these conditions. A registry of patients will reveal the natural history of the NIMBL diseases, and how effective current treatments are. By working in the laboratory and with individual patients, we hope to understand how and why these diseases occur and then identify potential elements we can target with drugs.
“We are particularly excited that the investigation of NIMBL diseases will not only improve the health and well-being of NIMBL patients and their families, but also lead to better treatments of much more common immune system disorders including lupus.”
The Manchester research team has also been given a grant of €176,000 by the European Leukodystrophy Association to pursue research into Aicardi-Goutières syndrome (AGS), one of the NIMBL group of diseases which destroys the brain in children and adults.
Professor Crow, who is Professor of Genetic Medicine at The University of Manchester and based in the department of Genetic Medicine at Saint Mary’s Hospital, Manchester, added: “The awards from the European Union and the European Leukodystrophy Association will allow for a step change in our studies of AGS and related disorders. By defining the natural history of these diseases and better understanding their cell biology, I am convinced that we can develop smart medicines for this devastating group of conditions.”
The NIHR Manchester Biomedical Research Centre was created by the National Institute for Health Research in 2008 to effectively move scientific breakthroughs from the laboratory, through clinical trials and into practice within hospitals to improve patient care. As a partnership between Central Manchester University Hospitals NHS Foundation Trust and The University of Manchester, the Biomedical Research Centre is designated as a specialist centre of excellence in genetics and developmental medicine
Source: The NIHR Manchester Biomedical Research Centre