SITraN is focused on translational neuroscience which means the goal of our research is to harness scientific developments emerging from experimental work in the laboratory and translate these insights into effective therapies and better outcomes for patients in the clinical care setting. We also bring what we learn from patients back to the laboratory to enhance our understanding of MND biology.
MND shares multiple commonalities with other common neurodegenerative diseases and is an excellent model for evaluating all forms of neuroprotective treatment. To facilitate optimum scientific cross-talk and to ensure that breakthroughs in one area can benefit research into related neurodegenerative disorders, SITraN hosts teams working on:
SITraN has the capability of taking therapeutic leads - based both on a strong in-house programme of neurobiology and international collaborations - into appropriate pre-clinical models, and through to novel therapies in humans. This is linked to a central role in the UK in the coordination of clinical trials in the field of neurodegenerative diseases (the DeNDroN network). In MND and SMA we have an international reputation based on integrating molecular science through to clinical studies.
Our partners from across the University of Sheffield as well as from national and international consortia bring further expertise in key areas required to speed up this critical path from the laboratory to the clinic.
SITraN brings together under one roof, state-of-the-art laboratories and equipment with a collaborative, multi-disciplinary environment including expertise in basic neuroscience, clinical neurology, neuropathology, computational biology, and clinical trials methodology. This multidisciplinary approach enables our team of basic scientists and clinical academics to work together to drive the translation agenda and to harness basic science to underpin drug discovery and clinical trials based on rational targets of proven preclinical effectiveness.
SITraN research teams have key skills in cellular and molecular biology, disease modelling, human neuropathology, viral vector technology, genetics, pharmacology, gene therapy, RNA processing, glial and mitochondrial biology, computational biology/bioinformatics and electrophysiology, together with neurology and clinical trials methodology. Other key areas under development within the institute are stem cell neurobiology and preclinical screening for new therapeutics in MND; these areas will complete the configuration of scientific staff and skills needed to focus successfully on our goal of developing therapies for MND and related neurodegenerative conditions.
The work of the SITraN research teams is based around the new genetic understanding of neurodegenerative diseases underpinned by the development of robust cellular and in vivo models of neurodegeneration (including transgenic mouse and zebrafish models), biosamples from human patients including the Sheffield Brain Tissue Bank, DNA, RNA, fibroblast, CSF banks, and a large repository of lymphoblastoid cell lines for several of the core diseases. Our key scientific strategy for discovery of promising drug targets is to model specific genetic subtypes of disease in parallel with detailed investigation of human biosamples. An iterative approach allows us to assess the likely relevance of new findings in the models by comparing them with the human disease state.
The dedicated research accommodation encompasses tissue culture suites, facilities for image analysis, live cellular imaging, confocal microscopy, laser capture microdissection, gene expression profiling by RNA microarray techniques and RNASeq, genetics, proteomics, histology, gene therapy, and small molecule drug screening. We have recently expanded our new generation gene sequencing technology with an Illumina Hi-Scan System. We also have an inter-faculty bioinformatics team embedded with the neurobiology teams within SITraN.