Late last year, the European Society of Gene and Cell Therapy and the British Society for Gene Therapy hosted a joint annual congress in Brighton, UK. The congress covered all aspects of gene and cell therapy and there were also satellite meetings which focused on how to get medicinal products licensed, as well as the way in which academic institutions and industry collaborate to develop gene therapy products.
Dr Suet-Ping Wong, working in the lab of Dr Richard Harbottle at Imperial College London, was nominated for the Fairbairn young investigator award and gave a talk on FLCN gene therapy using S/MAR vectors (for more information see our lab profile). The Harbottle lab have successfully developed vectors which express FLCN, and have shown re-expression of FLCN in UOK257 cells using these vectors. Additionally, the corrected cells have been xenografted into mice, with promising results. It is hoped that future in vivo experiments will continue to develop this therapeutic product as a potential treatment for BHD syndrome.
The difficulties facing the development of gene therapy products for rare diseases were highlighted by Dr Janneke de Wal from Amsterdam Molecular Therapeutics. The small population of affected individuals means data sets are small and large geographical areas need to be covered in order to gain enough participants for rare disease clinical trials. In Dr de Wal’s work developing Glybera, a gene therapy product for the rare disease lipoprotein lipase deficiency, more academics were involved than clinical trial participants.
Dr Marco Ranzani from the San Raffaele Telethon Institute for Gene Therapy in Milan gave another interesting talk on using insertional mutagenesis to identify cancer genes. Although insertional mutagenesis is seen as an adverse effect of gene therapy, it can be exploited for cancer gene discovery. Dr Ranzani showed that vectors which target and disrupt the Pten and Cdkn2a genes induced hepatocellular carcinoma (HCC) in mice models. Using this technique, the Braf, Fign, Sos1 and Dlk1-Dio3 genes were found to be associated with HCC development. Insertional mutagenesis using gene therapy could be a valuable technique to identify cancer-associated genes that could be potential therapeutic targets.
Information on upcoming meetings that may be of interest to BHD researchers can be found on the Conferences and Events page. More information on the upcoming 4th BHD Symposium, to be held in Cincinnati, USA, 28th-30th March 2012, will also be available soon.
www.bhdsyndrome.org – the primary online resource for anyone interested in BHD Syndrome.