Molecular Medicine and Gene Therapy

Faculty of Medicine | Lund University


Molecular Medicine and Gene Therapy

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The Division of Molecular Medicine and Gene Therapy is located at the Biomedical Center (BMC), Lund University, Sweden. Established as a joint venture between the Medical Faculty at Lund University and the Hematology Clinic at Lund University Hospital, our mission is to translate basic science to clinical applications.

Our research focuses on hematopoiesis, the continuous and dynamic process of blood cell formation. The laboratory consists of eight closely collaborating research groups that all share a common interest in investigating the properties of blood stem cells to eventually understand and treat hematological disorders.

Five of our researchers belong to the Hemato-Linné Excellence Linnaeus Research Environment funded by The Swedish Research Council and Lund University. Several of the groups are engaged in StemTherapy, a Strategic Research Area for Stem Cells and Regenerative Medicine that is also supported by The Swedish Research Council.


Shubhranshu Debnath
Shubhranshu Debnath

Congratulations to Shubhranshu Debnath and all co-authors, whose work "Lentiviral vectors with cellular promoters correct the anemia and lethal bone marrow failure in a mouse model for Diamond-Blackfan anemia" has been accepted in Molecular Therapy.

In this study, the authors demonstrate the feasibility of lentiviral-based gene therapy in a mouse model of Diamond-Blackfan anemia (DBA), a rare inherited bone marrow failure disorder. Using lentiviral vectors with cellular promoters, Debnath et al. cured DBA in a mouse model of the disease and improved the safety profile following integration as characterised by a lower risk of insertional oncogenesis. These findings support the potential of clinical gene therapy as treatment option for DBA patients in the future. 

Congratulations to all authors!

carolina guibentif

On May 11, Carolina Guibentif will defend her thesis entitled "Modelling Human Developmental Hematopoiesis".

Time and venue

May 11 at 9 am; Segerfalk Lecture Hall, BMC A10

Faculty opponent

Professor Nancy A. Speck, Perelman School of Medicine, University of Pennsylvania, USA 

Main supervisor

Associate Professor Niels-Bjarne Woods


Professor Jonas Larsson


kenichi miharada

Welcome to this month’s Stem Cell Talk, which will take place on Wednesday April 19th, starting at 14:45 with fika at Segerfalk Lecture Hall at A10. 

Speaker: Kenichi Miharada

Title: Stress response and management in hematopoiesis


Please welcome our new colleagues Emma Smith, Mayur Jain and Mitsuyoshi Suzuki, who recently joined the Division of Molecular Medicine and Gene Therapy.

Emma Smith will be working in Stefan Karlsson's group as a staff scientist, where she will be involved in a collaborative project that aims to develop gene therapy as treatment option for patients suffering from the rare genetic lysosomal storage disorder Gaucher's disease.

Mayur Jain joined Sofie Singbrant Söderberg's group as a postdoctoral fellow. During his postdoc project, Mayur will be elucidating disease contributing factors in myeloproliferative disease, and investigate how chronic anemia affects the ability of hematopoietic stem cells to provide a balanced blood production.

Mitsuyoshi Suzuki joined the Miharada lab from Juntendo University in Tokyo, Japan. During his postdoc project, he will be clarifying the role of bile acid in fetal hematopoiesis and liver development.

Welcome to our Divison!

carolina guibentif cell rep
Carolina Guibentif, first author of the study

Congratulations to Carolina Guibentif and all co-authors, whose work “Single-Cell Analysis Identifies Distinct Stages of Human Endothelial-to-Hematopoietic Transition" has been published in Cell Reports.

In this study, the authors provide a molecular characterisation of the developmental process of endothelial-to-hematopoietic transition (EHT) using human pluripotent stem cells (hPSCs)-derived cultures. Using a combination of flow cytometry and single-cell transcriptional profiling, Guibentif et al. demonstrate that hematopoietic differentiation potential is restricted prior to completion of EHT. These findings contribute to our understanding of how the first blood cells emerge in the human developmental context during their transition from endothelial to hematopoietic cells.

Congratulations to all authors!

This work lays the foundation for Carolina's thesis defence, which will take place on May 11th at 9 am. Carolina’s opponent will be Professor Nancy Speck from the Perelman School of Medicine at the University of Pennsylvania.

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