In most cases, the original disease-causing stem cells are effectively eliminated by the conditioning regimen (high-dose chemotherapy and/or radiation) used before the (autologous) transplantation, so they do not regenerate and cause further harm to the patient. However, in some cases, residual disease cells may survive the conditioning regimen and persist after transplantation, leading to disease relapse. This is more likely to occur in patients with aggressive or refractory disease or in those who receive a mismatched or haploidentical donor graft. To prevent disease relapse, post-transplantation maintenance therapy, such as immunosuppressive drugs or targeted therapies, may be used to eliminate residual disease cells. Additionally, close monitoring of the patient's blood counts and immune system function can help identify and manage any signs of disease relapse.
Re:First UK child to receive gene therapy for fatal genetic ...
by amelius on Friday February 17, 2023 @04:35PM
(#34840876)
Also, when cells replicate, will the new ones automatically be of the edited type? Or is it not that simple?
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Re:First UK child to receive gene therapy for fatal genetic ...
by ethanwillis on Friday February 17, 2023 @04:41PM
(#34840957)
In general it depends. If you modified existing blood cells genotype then it wont persist indefinitely. You'd need to modify the bone marrow. But then some cell lines work like you posed in your question.
Re:First UK child to receive gene therapy for fatal genetic ...
by MaryBall on Friday February 17, 2023 @04:50PM
(#34841067)
Because this therapy edits the stem cells that are found in the bone marrow, it essentially means that the disease is being fixed at the source. Bone marrow stem cells eventually turn into blood cells and immune cells. So if 100% of the cells have been edited, then the resulting cells they turn into will be the edited type.
Re:First UK child to receive gene therapy for fatal genetic ...
by janeway on Friday February 17, 2023 @05:24PM
(#34841446)
That is the goal but not that simple.