Professor Robert Carlisle, Course Director of the MSc in Nanotechnology for Medicine and Health Care, has been part of a research team exploring an aspect of gene therapy – lentiviral vectors (LVs) – and their potential to offer lifelong cures to many genetic disorders from a single dose.
The research was initiated by Prof Carlisle’s lab at the Institute of Biomedical Engineering (IBME), working with PhD student and first author Brannon Nicholls. Speaking recently on the Molecular Therapy Podcast, Prof Carlisle explained that his lab ‘has a focus on developing gene therapies for both treatment of solid tumors and also in vaccination strategies, and we have spent a long time optimising vectors, both viral and non-viral, and working out how to deliver those vectors better to their targets and we are very keen to take what we do from concept right through to the clinics.’
The challenge, however, of translating such therapies into human clinical applications, is that the typical models used in research often produce data that doesn’t reliably map onto the human body.
Their study addresses these limitations by using ex vivo human liver models – that is, human livers kept alive and stable outside of the body for many hours using specialised machinery. In this case, the machinery was a normothermic perfusion device, the development of which took place in Prof Carlisle’s lab. The four human livers in the study were kept in a state of continuous perfusion for up to 74 hours.
The paper ultimately represents an important opportunity in developing better models for drug delivery, and Prof Carlisle described their objective in this research: ‘to see if we can make something a bit more predictive and provide data that gives us a bit more assurance about the safety and efficacy of nanoparticles – gene therapy vectors – before we actually end up treating humans.’
Professor Robert Carlisle is Course Director of the MSc in Nanotechnology for Medicine and Health Care, offered jointly through the Department of Engineering and Oxford Lifelong Learning at the University of Oxford.
Learn more:
- Listen: the Molecular Therapy Podcast episode
- Read: Normothermic perfusion of human livers for profiling lentiviral vector pharmacokinetics and transduction
