PhD defence: Johannes Frasez Sørensen
Targeted gene editing investigated as a potential future treatment of acute myeloid leukemia
Info about event
Time
Location
Auditorium G206-142, Aarhus University Hospital
On Friday 6 November 2026 at 14:30, Johannes Frasez Sørensen defends his PhD dissertation entitled “Toward Gene Therapy in Acute Myeloid Leukemia with RUNX1::RUNX1T1: Clinical Rationale and Preclinical Development”.
Many patients with a particular form of acute leukaemia respond successfully to initial treatment, but the disease returns in approximately one in four. A new PhD project from Health at Aarhus University points towards a possible route to more targeted treatment. Drawing on nationwide Danish patient data, the project demonstrates the need for improved treatment and explores an advanced gene therapy based on CRISPR-Cas9 and lipid nanoparticles in laboratory studies.
The dissertation investigates a genetically defined subtype of acute myeloid leukaemia (AML) characterised by the RUNX1::RUNX1T1 fusion gene.
Acute myeloid leukaemia (AML) is an aggressive blood cancer in which immature leukaemic cells accumulate and interfere with the body’s normal production of blood cells. The course of the disease is strongly influenced by genetic alterations that divide AML into distinct subtypes. The RUNX1::RUNX1T1 fusion gene defines one such subtype and is generally associated with a favourable prognosis compared with other AML subtypes. Nevertheless, approximately 25% of patients experience relapse, which remains a major cause of treatment failure and highlights the need for improved treatment.
The dissertation investigates the disease from both clinical and translational perspectives. First, it examines the clinical characteristics and treatment courses of Danish patients with this type of AML. It then explores a new experimental treatment strategy in which lipid nanoparticles (LNPs) are used to deliver CRISPR-Cas9 directly to leukaemic cells, with the aim of inactivating the disease-driving RUNX1::RUNX1T1 fusion gene at the DNA level.
The studies demonstrate a clinical need for improved treatment options and provide preclinical evidence that targeted inactivation of RUNX1::RUNX1T1 may represent a promising therapeutic strategy. The findings therefore support the continued development and investigation of LNP-mediated delivery of intron-targeted CRISPR-Cas9 as a potential treatment for AML.
At the same time, important challenges must be addressed before the technology can be translated into treatment for patients. In particular, efficient systemic delivery to the disease-relevant leukaemic cells in the body and comprehensive evaluation of genomic safety will be essential for further clinical development.
The summary is written by the PhD student.
The defence is public and takes place in Auditorium G206-142, Aarhus University Hospital. Please see the press release for more information.
Contact
PhD student Johannes Frasez Sørensen
Mail: johannes.frasez@clin.au.dk
Phone: +45 21686092