Group Leader, The Roslin Institute
The Roslin Institute, University of Edinburgh
The most exciting thing that's happened this year in my research area:
The field of geen therapy is rapidly expanding with more potentially therapeutic products for a range of diseases that are now approved for clinical use or are in late phase clinical trials.
There are now licensed gene therapies for some forms of inherited blindness, some forms of leukaemia, some rare genetic diseases such as Spinal Muscular Atrophy and severe combined immune deficiencies.
My latest work:
I work as part of the UK Cystic Fibrosis Gene Therapy Consortium and, as the name suggests, we’re trying to develop gene therapy for cystic fibrosis. The Consortium has been running for almost 20 years now. It is a collaboration between the University of Edinburgh, the University of Oxford and Imperial College London.
Within the Consortium, our team at Roslin is using sheep as a model to study gene delivery to the lung in people. We have been involved in a lot of preclinical testing of gene therapy vectors, which we then try to develop and optimise. Over the years, our gene therapy vectors have even gone as far as clinical trials in cystic fibrosis patients with some success.
In the past few years, we’ve been looking at using a new viral-based vector, giving us a very optimistic view for the future. This viral based vector, an SIV vector which can cause disease in primates, is similar to HIV. It is essentially the same kind of virus therefore it has to be modified to prevent it from being harmful.
We have been working on these viral-based methods for a while and it seems that they are more efficient than the non-viral systems used previously. The advantage of the viral mechanism is that SIV integrates into the genome of patients receiving treatment.
We’re quite optimistic and we’ve got funding in place from the Wellcome Trust and the Department of Health through the Health Innovations Challenge Fund that allows us to do clinical trial. We’ve also recently partnered up with a pharmaceutical company, which allows us to do a much more comprehensive program and hopefully get to clinic quicker. We really are quite optimistic for the future as this partnership may really provide us with a lot more resources, which we really need to be able to make this a viable treatment for patients.