Catholic University of Leuven, Belgium Genome editing for Wolfram syndrome: Although rare individually, genetic disorders collectively constitute a common health problem. As the cause of these diseases is a defective gene, gene therapy would be able to resolve all of these disorders. Wolfram syndrome is a genetic disorder, with the ultimate symptoms of Diabetes, blindness […]
Update January 26, 2024: Dear Friends, Thank you sincerely for your invaluable support of our collaborative study alongside Prilenia Therapeutics. As you are aware, our mission to find a cure for Wolfram syndrome involves three crucial steps: Step 1: Slowing down the progression of the disease through the use of oral medications. Step 2: Halting […]
Update March 5, 2024 Our focus is to discover preclinical Liraglutide and the rest of the GLP1 receptor agonists available on the market to help stop the progression of Wolfram syndrome. Moreover, we are looking into a new generation of GLP1 and GIP co-agonists for the same reason. We are also developing gene therapy against […]
University of Cambridge, United Kingdom We are currently focusing our efforts on the following research areas. Disease progression and biomarkers We are collecting a comprehensive range of ophthalmological data on individuals who have been confirmed to carry pathogenic WFS1 mutations, including high-resolution optical tomography tomography imaging and visual electrophysiology. We want to carefully document the […]
Institute of Biomedicine and Translational Medicine, University of Tartu, Tartu, Estonia Professor Kaasik and his colleagues from the University of Tartu (Estonia) are currently searching for small molecular compounds to improve the function of endoplasmic reticulum that could have potential for the treatment of Wolfram Syndrome. They have identified several compounds with the ability to improve endoplasmic calcium homeostasis and neuronal […]
Washington University School of Medicine Drs Hershey, Marshall and the rest of the Wolfram Research Clinic team have been busy analyzing data that has already been collected and trying to plan for the next round of data collection during the current pandemic. We hope to start remote data collection (surveys, zoom interviews) this month, with […]
ULB Center for Diabetes Research, Brussels, Belgium GLP-1 analogs, such as liraglutide, exenatide and dulaglutide among others, are used to treat type 2 diabetes. These drugs are known to promote pancreatic beta cell function and survival, may cross the blood brain barrier, and might have potential beneficial effect on neurons and retinal cells. GLP-1 analogs […]
In my lab, we are developing concomitantly two therapeutic strategies: a pharmacological approach and a gene therapy. To achieve these goals, we are working with suited animal models: two transgenic mouse lines and one zebrafish line. One mouse model and the zebrafish line are deficient for Wolframin, the protein responsible for Wolfram syndrome type 1. […]
Especialista en Medicina Familiar y Comunitaria Coordinadora del Equipo multidisciplinar del Síndrome de Wolfram Español Below are the most important activities led by Dr. Gema Esteban Bueno as head of the Spanish Clinical Wolfram Syndrome Team and scientific head of the Spanish Association for Research and Assistance to Wolfram Syndrome (AEIASW).. It also lists the […]
University of Birmingham, United Kingdom
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