Pediatric Cell and Gene Therapy
Our team is working in pediatric hematology and oncology in the field of hematopoietic stem cell transplantation. By utilizing various cell and gene therapy approaches, we aim to develop novel gene therapies for β- hemoglobinopathies (β-thalassemia, sickle cell disease), cancer immunotherapy, and rare diseases including metachromatic leukodystrophy, as well as immunodeficiencies. Our technologies include lentiviral and Adeno-associated virus gene transfer, mRNA therapeutics, adoptive immune cell therapy, genome editing and gene correction using CRISPR / Cas9 system. For more detailed information, see our homepage at UKT (link below).
List of GRT projects
- Gene correction of human b-thalassemic hematopoietic stem cells using CRISPR/Cas9 system
- Hematopoietic stem cell gene therapy with Cas9 mRNA and AAV6 vector in metachromatic leukodystrophy
- Improvement of CAR T-cell and CAR NK-cell anti-leukemic effector functions using CRISPR/Cas9 technology
- Antibody optimization in CHO-S cells using CRISPR/Cas9 gene editing for enhanced anti-tumor activity
- Using CRISPR/Cas9 system and prime editing for the treatment of IL2RG immunodeficiency
- Improvement of cell fitness after CRISPR/Cas9 electroporation by anti-ROS compounds
GRT expertise
Hematology, oncology, hematopoietic stem cell transplantation, b-hemoglobinopathies, metachromatic leukodystrophy, ex vivo gene editing
Main GRT methods applied in the lab
CRISPR/Cas system, AAV based gene correction, lentiviral gene therapy, mRNA production and delivery, transfection of primary hematopoietic stem cells, CAR T-cells, CAR NK cells, CliniMACS Prodigy, on-/off target analysis, prime editing, digital droplet PCR