Atherosclerosis is the main underlying cause of cardiovascular disease (CVD) and due to its multifactorial natural is often studied in animal models. Here the aim is to develop an innovative ex vivo system to study the atherosclerotic disease process based on the actual human atherosclerotic plaque. In line with the Transition to Animal-free Innovation (TPI), the aim is to develop a model that generates data on the effect of novel drugs that is highly translatable to the effect of the novel drug in the cardiovascular patient, while at the same time minimising the number of laboratory animals used. Moreover, the aim is to utilise this model to discover new biomarkers of successful treatment, which could expedite the transition of CVD therapeutic into clinical trial.
The IMAGEN consortium aims to improve diagnostics of patients with ciliopathies and tubulopathies, enhance quality of care, and translate genetic findings to personalised care by testing pharmacological treatment approaches based on patient-derived disease models.
The RBD‐CURE project will develop innovative gene therapy approaches for patients with rare bleeding disorders. The unique expertise of Sanquin on bleeding disorders together with the advanced gene therapy strategies developed by SanaGen and the Netherlands Institute for Neurosciences aims to provide a permanent cure for patients with rare bleeding disorders.
Hepatocellular carcinoma is the most frequent liver malignancy and the fourth most common cause of cancer-related death worldwide. There is a clear clinical need for accessible and reliable parameters to identify high-risk populations in order to decrease the high mortality rates of hepatocellular carcinoma.
In muscle diseases, muscle fibers lose their capacity to contract, resulting in loss of mobility and respiration. In this project advanced 3D models of muscle diseases in vitro will be generated and the effects will be investigated on the smallest contractile unit present in muscle cells using highly sensitive force measurements.
The immune system has potent mechanisms with which it deals with intruding viruses, bacteria and tumors, protecting us from disease. However, tumors contain mutations leading to resistance to the immune system. The NextIO project aims to develop a medicine that restores the sensitivity of the cancer to the immune system.
The aim of this project was to develop methodologies for robust, and safe in vivo gene editing in skeletal muscle tissue. The project has provided a valuable tool set for researchers around the world to perform gene targeting in skeletal muscle, enabling the development of personalised gene therapies for genetic muscle disease and metabolic diseases.
Acute myeloid leukemia (AML) is a form of blood cancer that is still difficult to cure and better therapies against AML are clearly needed. As a continuation of a long-standing collaboration between the Schuringa lab at the UMCG and Janssen will exploit potential novel targets against leukemic stem cells.
Increasing evidence suggests that dynamic changes in the transcriptional state of specific cell-types plays a key role in determining disease outcomes. Here, cell-type specific changes will be detected in tuberous sclerosis complex cortical tubers and assessed if these cell-type changes are involved in the pathogenesis of epilepsy.
The aim is to improve vaccines by including viral innate immune antagonists that are co-expressed with the antigen to boost vaccine efficacy.