AAV gene therapy
We are involved in a number of AAV gene therapy projects ranging from preclinical studies of continuous L-DOPA delivery for the treatment of Parkinson’s to the development of new AAV capsid variants with improved biodistribution abilities. In this context, we have established collaborations with investigators at King’s and other UK universities and have teamed up with industrial partners.
We have recently been awarded a Pfizer Rare Disease Consortium Grant to develop an AAV vector platform for the treatment of rare diseases. This award is also sponsoring a new line of research in my laboratory that is designed to apply insights into the basic understanding of the virus to help overcome the challenges of production for clinical use.