Gene Medicine Research Group – Nuffield Division of …

Introduction

The Group is led byProf Deborah Gill andAssociate ProfSteve Hyde and forms one third of the UK Cystic Fibrosis Gene Therapy Consortium. The main aim of this Consortium of scientists and clinicians, is to make gene therapy for Cystic Fibrosis (CF) lung disease a clinical reality. Early Phase I clinical studies have already demonstrated proof of principle for CF lung gene therapy leading to correction of the CF genetic defect in the epithelium lining the nose and lung of CF patients. The clinical development of new gene therapy formulations for CF and other lung diseases is underway. The cationic lipid GL67A complexed with a novel CFTR expression plasmid has been aerosolized to the lungs of patients with Cystic Fibrosis and has been shown to be sufficiently safe for long-term evaluation. We can now deliver monthly doses of this formulation, over a 12 month period, to determine if this leads to clinical benefit. In parallel we are interested in developing viral vectors for gene delivery to the lungs. We are currently developing a new viral gene therapy vector using Lentivirus pseudotyped with envelope proteins that permit efficient entry into lung cells.

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Gene Medicine Research Group - Nuffield Division of ...