Lung Gene Therapy
2012 – 2015 Research Technician
Validation of Human Ex Vivo Models For Airway Gene Transfer Duration
Doctor Uta Griesenbach
We are interested in the development of lung gene therapy for cystic fibrosis (CF) and have ~ 20 yrs experience in the field. CF is a fatal disease caused by a faulty gene (CF gene).
The concept of gene therapy is simple. One “just” has to put a normal gene back into the cells. However, this requires the normal gene to be combined with a carrier to help transport the gene into the cells.
The UK CF Gene Therapy Consortium (www.cfgenetherapy.org.uk) has developed ways to introduce the CF gene into cells and we are currently preparing for the world’s largest CF gene therapy trial program (approx. 130 subjects, due to start in early 2012), which will assess if repeated administration of the CF gene, combined with the most efficient current delivery system, can improve CF lung disease.
In parallel we have developed a new way of delivering the gene (a virus), which holds great promise for airway gene therapy and has won the 2011 MRC Translational Research Award and Best Respiratory Therapeutic Innovation in the Medical Futures Awards. We plan to develop this virus further to progress into clinical trials around 2014/15.
It has been shown that currently available animal models are comparatively poor predictors of how well the gene can be delivered and how safe it is. Recently we have gained access to several novel human lung models which are likely to mimic the human lung considerably better. Comparing our current best gene delivery methods in these models, and in our ongoing clinical trial, will give us the unique opportunity to compare directly efficiency and safety and, thus, validate these novel models. In addition, we will be able to assess our new delivery system in these human models.
These studies will help both us, and many others, to more quickly progress from pre-clinical research into clinical trials and to reduce the number of animals required for pre-clinical research.