![]() ‘Our aim is to develop a method for how society can deal with these therapies. But the price tag remains unclear for the long term.’ Staal recently received a grant to study this. ‘Since the therapy is still being used for research purposes, it does not cost the patient anything. ‘This means we are not dependent on the pharmaceutical industry,’ says Staal, ‘which reduces the costs of the therapy to some extent.’ However, like other gene therapies, this treatment is still very expensive. LUMC is one of the few hospitals in the Netherlands with a licence to genetically modify cell products. Stem cell gene therapy is produced in a special laboratory. ![]() Further research will show whether this therapy will become the new standard for SCID in the future. ‘We avoid these problems in stem cell gene therapy because patients are their own donors,’ says Lankester. For example, a good donor isn’t always available and there are risks to transplanting another person’s cells. However, the current treatment, namely stem cell transplantation, has a number of disadvantages. ![]() Without treatment, children with SCID often die before the age of one. Staal and Lankester expect the patient to be cured for life with this complex yet one-off treatment. The virus is therefore just the wheelbarrow that brings the gene to the right place.’ The repaired cells were then transferred back into the patient, who was allowed to leave the hospital within a month. ‘We only use the virus’s ability to build its own genome in its host’s DNA and remove all its other properties. ‘We use a crippled virus for this,’ explains Frank Staal, Professor of Stem Cell Biology. Stem cells were taken from the patient three months after birth and a good copy of the gene, in this case the RAG1 gene, was subsequently inserted into the DNA of these cells. ‘The baby has undergone the treatment without any problems and its immune system is responding well to the usual vaccinations for newborns. ‘The corrected stem cells grew into a functioning immune system in the patient’s body,’ says Arjan Lankester, Professor of Pediatrics and Stem Cell Transplantation. Stem cell gene therapy can repair this error. Due to a DNA error in their blood stem cells, SCID patients do not produce immune cells and their body cannot defend itself against infections. The patient is a baby who is just a few months old and suffers from SCID.
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