Blood Research Theme
Our Blood research team is developing new gene therapies for inherited conditions affecting the blood and immune system, with an initial focus on rare genetic disorders that leave patients particularly vulnerable to infections, including those caused by the Epstein-Barr virus (EBV).
The team is investigating how gene editing technologies can be used to correct disease-causing changes in a patient's DNA. This involves collecting blood cells from patients, precisely modifying their genetic material in the laboratory, and returning the modified cells to the body. The aim is to restore normal cell function and address the underlying genetic cause of disease, rather than simply treating its symptoms.
Alongside this research, the team is developing the methods needed to translate these experimental approaches into potential clinical treatments. This includes investigating how gene-edited cells can be produced reliably, tested for safety and effectiveness, and manufactured to the standards required for use in patients.
By establishing and refining these approaches, the team hopes to create a foundation for developing gene therapies for a wider range of inherited blood and immune disorders.
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
Beyond innovation: why rare disease gene therapy needs a reset
Health economic evaluations of genomic newborn screening: Approaches by studies within the international consortium on newborn sequencing
Personalized CRISPR therapies could soon reach thousands — here’s how
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
Liz Brown
Postdoctoral Researcher
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
Rui Chen
Postdoctoral Researcher, Newcastle University
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
Roman Doll
DPhil Student
Karin Englehardt
Postdoctoral Researcher, Newcastle University
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
Havinder Hara
Clinical Operations Manager
Anna-Sophie Haselon
DPhil Student, University of Oxford
Aga (Agnieszka) Kubat
Senior Clinical Project Manager
Hangpeng Li
DPhil Student, University of Oxford
David Newman
Postdoctoral Researcher
Aideen O'Neill
Postdoctoral Researcher
Bora Ozcan
DPhil Student, University of Oxford
Louis Peters
DPhil student
Neve Richardson
Research Assistant
Viviana Salinas-Rios
Director CRISPR Cures Technology Core
Giorgia Santilli
Associate Professor
Ina Schim van der Loeff
MRC CoRE Blood Theme Co-Lead
NIHR Academic Clinical Lecturer in Paediatrics, Newcastle University
Kim Sharp
Postdoctoral Researcher in Genomics and Genome Editing
Jarmila Spegarova
Postdoctoral Researcher, Newcastle University
Zohar Steinberg
PhD student
Taylor Hudson
Graduate Student
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
Ye Wei
Postdoctoral Researcher
Daniel Willmott
Research Assistant in Genome Editing, University of Oxford
Weijiao Zhang
Research Associate