Related Experiment Video
Updated: Nov 29, 2025

Generation of Multivirus-specific T Cells to Prevent/treat Viral Infections after Allogeneic Hematopoietic Stem Cell Transplant
Published on: May 27, 2011
[Treatment of monogenic disorders with viral transduced haematopoietic stem cells]
Marianne Segelcke Ifversen1, Tania Nicole Masmas, Brian Kornblit
1Marianne.Ifversen@regionh.dk.
Abstract:
Infusion of ex vivo transduced haematopoietic stem cells (HSC) has emerged as a promising new treatment of certain monogenetic disorders. Since early clinical studies on patients with severe combined immune deficiency were halted due to de novo leukaemia, the technology has matured. Thus, treatment of transfusion-dependent thalassaemia and adenosine deaminase deficient severe combined immunodeficiency by using lentiviral vectors for gene correction of autologous HSC can induce expression of the deficient protein and thus potentially cure the patients. The review summarises recent advances allowing for clinical implementation of the treatment in Denmark.
Related Concept Videos
Bone Marrow Sampling and Transplants
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
Stem Cell Therapy for Tissue Regeneration
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
Gene Therapy
Multipotency of Hematopoietic Stem Cells
iPS Cell Differentiation

