Paediatric Haematology, Oncology and Stem Cell Transplantation
Gene therapy
Gene therapy is understood to be the targeted modification of genes in human cells, curing the causes of a hereditary disease directly. Unlike conventional forms of treatment, gene therapy is not a symptomatic therapy.
Unlike conventional forms of treatment, gene therapy does not address specific symptoms.
First successful gene therapy in Germany
Our department was the first in Germany to successfully treat a patient with thalassaemia using CRISPR/CAS9-modified stem cells.
The clinical successes achieved to date in these CRISPR/CAS9 gene editing studies for sickle cell disease and transfusion-dependent thalassaemia represent a revolutionary advance in the treatment of congenital haematological disorders. Thanks to this pioneering work, they are now available as a therapy option covered by health insurance for certain diseases.
If you are interested, please feel free to contact us for a detailed consultation.
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Somatic gene therapy involves harvesting the patient's own (autologous) haematopoietic stem cells. These cells are provided with a modified copy of the defective gene in the laboratory.
The corrected cells are then returned to the patient after what is known as myeloablative chemotherapy.
Genome modifications carried out as part of so-called somatic gene therapy are not passed on to offspring.
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There are currently few effective treatments for the large group of hereditary (genetic) diseases. These include thalassaemia and sickle cell disease, the most common genetic disorders worldwide.
Our team has clinical experience in the gene therapy approach for patients with congenital haematological disorders such as sickle cell disease and thalassaemia.
The production of foetal haemoglobin is reactivated in these diseases by means of ‘gene scissors’.
Foetal haemoglobin is not affected by genetic changes, eliminating the need for transfusion therapy or the symptoms of sickle cell disease.
