SIDB is ideally positioned to exploit the unprecedented opportunities provided by the genomics revolution. In particular, SIDB uses new technologies such as gene-editing, optogenetics and imaging. These are complementary to our clinical expertise in cohort building and deep phenotyping at the University of Edinburgh. We will take advantage of these to facilitate experimental medicine and clinical trials.
As well as large-scale approaches with standardised research protocols across animal models, we use bespoke mechanism-based approaches that require either cutting-edge technologies or proof-of-concept studies in individual models.
We exploit our wide-ranging strengths in fundamental research to pursue both bottom-up and top-down approaches. The bottom-up approach targets either genetic causes using novel gene-editing strategies and virus-based gene delivery systems, or the molecular/biochemical pathways directly affected by the genetic alteration. The complementary top-down approach targets the circuit and cellular deficits in an attempt to directly restore normal brain physiology.
Using both approaches, allows us to not only devise novel interventions, but to rigorously test them before selectively progressing potential treatments to the clinic.
We have brought together neuroscientists from the University of Edinburgh studying the brain at the level of molecules, cells, circuitry and behaviour. This team of scientists will exploit the wealth of knowledge obtained from large-scale genetic studies to model genetic alterations from gene candidate lists in rodent and human models.
Genetic disorders may be notionally treated in either of two ways: by rectifying the primary mutational cause; or by attacking the secondary consequences of the genetic lesion. SIDB focuses on devising both primary interventions and secondary therapies that target the downstream effects of genetic errors.
The brain and nervous system is an incredibly complicated structure. Understanding how it works is essential to appreciating how this can go wrong and what interventions might be effective in delivering relief. While our researchers are committed to the principals of the 3Rs and so, where possible, they develop and use experimental approaches that Replace, Reduce or Refine animal use, there are instances where there are no alternatives to the use of animals.