Blood Research Theme
The Blood Theme is working to develop a robust, end-to-end pipeline for gene editing therapies.
The initial focus is on primary immune deficiencies associated with Epstein-Barr virus (EBV).
Our initial work explores ex vivo base editing applications: blood can be taken from patients, treated with our therapy in a laboratory, and tested before being transfused back into the patient.
This work is designed to de-risk the complex regulatory and manufacturing pathways for all future MRC CoRE gene editing therapies: creating a comprehensive template for engagement with bodies like the MHRA and FDA, patient involvement, and clinical readiness that can be applied to a wider range of blood and immune disorders in the future. The Blood Theme will also develop a Genomic Multi-disciplinary Team (MDT) to decide on candidate target genes, and source patients and clinical data, as well as a Process Development MDT to align critical methodologies.
Research projects
Automated cell culture and screening at scale
Using the CellXpress.ai platform for faster and more standardised testing of potential therapies
EBV-handling disorders
Ex vivo gene editing for five target genes
Theme publications
Mapping chromatin structure at base-pair resolution unveils a unified model of cis-regulatory element interactions
Team
Professor Claire Booth
Mahboubian Professor in Gene Therapy and Head of Department of Paediatric Immunology, UCL
Consultant in Paediatric Immunology, Great Ormond Street Hospital
Professor Ronjon Chakraverty
MRC CoRE TG Blood Theme Lead
MRC CoRE TG Patient and Public Involvement and Engagement Lead
Professor of Haematology and Attending Consultant Physician, University of Oxford
Dr Rui Chen
Postdoctoral Researcher, Newcastle University
Dr James Davies
MRC CoRE TG Blood Theme Co-Lead
Associate Professor of Genomics, University of Oxford
Anne Marie Delaney
DPhil Student, University of Oxford
Nick Denny
DPhil Student, University of Oxford
see also https://github.com/ndrdenny
Dr Karin Englehardt
Postdoctoral Researcher, Newcastle University
Dr Helen Griffin
Research Associate, Newcastle University
Professor Sophie Hambleton
MRC CoRE Blood Theme lead
Professor of Paediatrics & Immunology, Newcastle University | clinical paediatric immunologist at Great North Children's Hospital, Newcastle
Joseph Hamley
DPhil Student, University of Oxford
Anna-Sophie Haselon
DPhil Student, University of Oxford
Hangpeng Li
DPhil Student, University of Oxford
Bora Ozcan
DPhil Student, University of Oxford
Dr Ina Schim van der Loeff
MRC CoRE Blood Theme Co-Lead
NIHR Academic Clinical Lecturer in Paediatrics, Newcastle University
Dr Jarmila Spegarova
Postdoctoral Researcher, Newcastle University
Professor Fyodor Urnov
MRC CoRE TG UC Berkeley Site Lead
Professor of Molecular Therapeutics
Director, Technology and Translation, Innovative Genomics Institute, University of California, Berkeley
Director, IGI - Danaher Beacon for CRISPR Cures
Director, Center for Pediatric CRISPR Cures
Daniel Willmott
Research Assistant in Genome Editing, University of Oxford