Trial lecture
The candidate will first give a trial lecture on the following topic: 'Haemopoetic stem cells as cause of cancer'
Time and place: Nov. 26, 2025 10:15 AM, Nucleus, Bikuben, Kristine Bonnevies hus
Main research findings
In this study, Katariina Mamia and colleagues developed an efficient and safe gene editing platform that can correct genetic “typos” in human T cells. This platform can correct single-gene mistakes in T cells from patients with rare diseases affecting the immune system, also known as Inborn Errors of Immunity (IEI).
Currently, some IEIs can be treated with stem cell transplants from healthy donors, but this approach carries serious risks and is not an option for everyone. To address this unmet need, Katariina and colleagues used CRISPR/Cas9 gene editing technology to modify the patients’ own T cells instead. In this approach, T cells are isolated from blood samples, corrected in a laboratory – and one day, transplanted back into the patients.
The platform achieved up to 80% correction efficiency and was shown to improve T cell functions in the corrected T cells. Extensive safety testing detected no harmful effects or unwanted genetic changes within the methods used.
This study presents a first-generation T cell correction platform that can repair various single-gene defects with one workflow. This study presents a first-generation T cell correction platform that can repair various single-gene defects with one workflow. It represents an early step towards clinical translation of gene therapies for IEI patients who currently have limited treatment options.
Adjudication committee
Prof. Claire Booth, Infection, Immunity & Inflammation Department, University College London
Prof. Judith Zaugg, Department of Biomedicine, University of Basel
Prof. Andreas Brech, Oslo University Hospital & Department of Biosciences, University of Oslo
Chair of defense
Group leader Bernd Thiede, Department of Biosciences, University of Oslo
Supervisors
Head of Group Emma Maria Haapaniemi, NCMBM - Norwegian Centre for Molecular Biosciences and Medicine, University of Oslo
Prof. Johanna Olweus, Oslo University Hospital & Institute of Clinical Medicine, Faculty of Medicine, University of Oslo
Prof. Jorrit M. Enserink, Department of Biosciences, University of Oslo