Trial lecture
The candidate will first give a trial lecture on the following topic: 'The history and current state-of-the-art for gene therapies using blood stem cells'
Time and place: Jan. 14, 2025 10:15 AM, Nucleus, Bikuben, Kristine Bonnevies hus
Main research findings
We show that gene editing can correct a rare immune disorder called DADA2. Using CRISPR, a “molecular scissor,” we fixed a single-letter error in the ADA2 gene with high precision (up to 80%) and tested adding a healthy copy of the gene as a broader solution. Edited blood stem cells stayed healthy, made balanced blood cell types, and worked in mice.
DADA2 often starts in childhood and can cause serious illness. Current treatment can be a stem cell transplant from a donor, but donors are scarce and the procedure can fail. Our work points to a patient-tailored therapy that uses a person’s own stem cells, and a near-universal option that could help many patients regardless of their exact mutation.
The tools are powerful and flexible, and they could change how we treat rare genetic diseases. But cutting DNA can introduce risks, such as unwanted changes. Therefore, we tested safety in several ways and found encouraging results, yet more work is needed to ensure long-term safety before clinical use.
Adjudication committee
Prof. David Kent, Department of Biology, University of York
Prof. Julia Skokowa, Department of Hematology, Oncology and Clinical Immunology, University Hospital Tuebingen
Assoc. Prof. Pierre Chymkowitch, Department of Biosciences, University of Oslo
Chair of defense
Prof. Eivind Valen, 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. em. Oddmund Bakke, Department of Biosciences, University of Oslo
Prof. Espen Melum, Oslo University Hospital & Institute of Clinical Medicine, University of Oslo
Assoc. Prof. Rasmus O. Bak, Department of Biomedicine, Aarhus University