Research groups
Websites
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The UK Cystic Fibrosis Gene Therapy Consortium
The UK Cystic Fibrosis Gene Therapy Consortium
DPhil Supervisor Profile
Gill Group – Development of Gene Therapy and Gene Editing for Lung Disorders
Deborah Gill
PhD, BSc (Hons)
Professor of Gene Medicine
Deborah Gill is based in the John Radcliffe Hospital in Oxford where she is Professor of Gene Medicine and Co-Director of the Gene Medicine Research Group.
Deborah completed her PhD in molecular microbiology at the University of Warwick, studying cell division proteins in E.coli, during which she discovered the defining bacterial member of the ABC (ATP-Binding Cassette) superfamily of proteins. In 1990, Deborah moved to the University of Oxford, to undertake post-doctoral research under the mentorship of Professor Chris Higgins at the Weatherall Institute for Molecular Medicine, investigating human ABC proteins including the Multi-Drug Resistance p-glycoprotein and CFTR, the protein responsible for Cystic Fibrosis (CF).
Together with Dr Steve Hyde, Deborah's research began to evaluate gene therapy for genetic diseases, focusing on a potential treatment for Cystic Fibrosis lung disease. Two clinical trials in CF patients were undertaken in collaboration with Sir Martin Evans, and Professors Bill Colledge and Alan Cuthbert at Cambridge University, demonstrating proof of principle for non-viral (plasmid-based) CF gene therapy in the upper airways.
In 2001, Deborah was a founding member of the UK CF Gene Therapy Consortium, joining together the Gene Medicine Research Group, University of Oxford; the Centre for Molecular Medicine and Roslin Institute, University of Edinburgh; and, the Department of Gene Therapy, National Heart & Lung Institute, Imperial College London. This consortium of scientists and clinicians continues to work towards making gene therapy a reality for patients with CF. The Consortium completed the world's largest gene therapy trial - a phase llb study involving 136 participants - delivering an aerosol of non-viral gene therapy at monthly intervals for 12 months. The study showed a clinically relevant improvement in lung function in those receiving gene therapy, compared with those receiving a placebo treatment. However, more efficient gene delivery strategies are needed.
The success of gene therapy to introduce genes into cells and gene editing to correct genetic mutations, ultimately depends on efficient delivery of genetic material to human cells. Much of Deborah's current research is focused on development of Lentiviral and AAV vectors for translation of new gene therapies to the clinic. In addition to targeting genetic lung diseases Deborah's research group is evaluating whether the airways can be used as a 'protein factory' to make therapeutic proteins and anitbodies.
As well as undertaking translational research, Deborah is involved in many aspects of graduate student mentoring and training. She has supervised many DPhil and Masters students to completion of their post-graduate degrees and is currently the Chair of Oxford University's Graduate School Committee for the Medical Sciences Division.
Key publications
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Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.
Journal article
Alton EWFW. et al, (2015), Lancet Respir Med, 3, 684 - 691
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CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression.
Journal article
Hyde SC. et al, (2008), Nat Biotechnol, 26, 549 - 551
Recent publications
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Lessons learned from lung and liver in-vivo gene therapy: implications for the future
Journal article
van Haasteren J. et al, (2018), Expert Opinion on Biological Therapy, 18, 959 - 972
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The murine lung as a factory to produce secreted intrapulmonary and circulatory proteins.
Journal article
Paul-Smith MC. et al, (2018), Gene Ther
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Lentivirus GM-CSF gene therapy for autoimmune pulmonary alveolar proteinosis
Conference paper
Lund-Palau H. et al, (2018), HUMAN GENE THERAPY, 29, A2 - A2
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Large-scale, high-throughput production of lentiviral vectors for multiple disease applications
Conference paper
Ashworth RC. et al, (2018), HUMAN GENE THERAPY, 29, A6 - A6
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Use of Pseudotyped, Replication-Deficient Influenza to Facilitate Testing of Gene Delivery Strategies for Treatment of Highly Infectious Diseases
Conference paper
Tan TK. et al, (2018), MOLECULAR THERAPY, 26, 242 - 243