RNA - Genetherapys

Identifying New Biomarkers to Treat Type 1 Diabetes

National Center Researchers Offer Tailored Precision-Medicine Approach to Treat Type 1 Diabetes

Funded through the National Center for Gene Therapy and RNA Drugs, the high-impact study published by Spoke 5 researchers at the University of Siena in Cell Reports Medicine identifies biomarkers for the personalized treatment of type 1 diabetes.

As an autoimmune disease once known as juvenile diabetes, type 1 diabetes mellitus (T1DM) results in chronic hyperglycemia, lifelong insulin therapy, and the manifestation of diabetic complications. As the direct cause of this autoimmune disease is unknown, genetic and environmental factors distract the insulin-producing cells in the pancreas. With peak onset during childhood, an estimated 80,000 children develop the disease each year. Thanks to the research team, led by Prof Francesco Dotta of the University of Siena, new personalized treatment strategies may improve the quality of life and the management of those suffering from the disease.

As the Head of the Department of Medicine, Surgery, and Neuroscience at the University of Siena, and Director of Diabetes and Clinical Nutrition Units at Policlinico University Hospital in Siena, Prof Dotta oversaw the international collaborative work. Beyond the National Center, additional support came from the European Union as part of the INNODIA project. Using advanced RNA sequencing technology that only requires a few microliters of blood, researchers identified a class of small noncoding RNAs, called circulating microRNAs, which could offer valuable diagnostic and prognostic tools for T1DM.

The first author of the study and Associate Professor of Laboratory Medicine at the University of Siena, Prof Guido Sebastiani explains,

“MicroRNAs are small RNA molecules that regulate many biological processes, including those linked to the onset and progression of type 1 diabetes. Our work analyzed the microRNAs present in the blood gathered from a large cohort of those affected by this disease, revealing a set of circulating microRNAs belonging to a specific region of chromosome 14, known as 14q32 that alters susceptibility to type 1 diabetes.

These microRNAs have allowed us to identify two subgroups of individuals with recent onset of type 1 diabetes, defined as Cluster A and Cluster B. Subsequent analyses on another group of people have confirmed this distinction. Our work unveiled those in Cluster B have an increased expression of a specific group of microRNAs that show better glycemic control and a different blood immunological profile.”

Prof Dotta concludes,

“Our research identifies potential biomarkers that could change the way we understand and treat type 1 diabetes. This discovery, combined with the evidence that it is also a heterogeneous disease, offers hope for more targeted and personalized therapies. I also emphasize that after more than a century since the discovery of insulin, the development of therapeutic strategies has just begun. This is the right step towards protecting the beta cells in the pancreas and blocking cells in the immune system before the onset of the disease.

The US Food and Drug Administration has already approved the first monoclonal antibody drug directed against T lymphocytes. In this context, the personalized medicine approach using specific circulating microRNAs may represent excellent candidates to identify and treat subgroups with the appropriate therapeutic strategy. Our research suggests that it may be possible to use a simple blood test to measure circulating microRNAs and identify two distinct subgroups of those with type 1 diabetes. Furthermore, we can apply new personalized treatment strategies, based on specific immunological and clinical characteristics, to improve the diagnosis and the management of the disease.”

Written by

The Foundation’s editorial staff

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