11/6/2022 0 Comments Gene therapy for scid![]() ![]() GENE THERAPY FOR SCID TRIALIf the immune system is not too bad, then the trial might not be a good fit.Īlso, most older children or young adults who have waning immunity or poor function after stem cell transplant may have more organ damage because of infection in their lungs or gastrointestinal tract. In addition, the immune system must be sufficiently defective so that the risk of going through the gene therapy is balanced out by how bad the immune system is. ![]() Because gene therapy is currently only offered in the context of a clinical trial, an older child or young adult who has already had a stem cell transplant must meet the criteria for that study, just like a newly diagnosed infant would. How can parents know if gene therapy is appropriate for an older child or young adult who has had one or two stem cells transplants already for SCID?Ī. A child might need a certain level of infection control or body organ function to participate.Īlso, because the outcomes of the matched sibling donor in hematopoietic stem cell transplant, or HSCT, (also known as bone marrow transplant) are so excellent, all of the current gene therapies are available only to those with no matched sibling donor. Therefore, the decision about whether gene therapy is appropriate or can be offered depends on each trial’s eligibility criteria for the child. Right now, all of the gene therapy that we listed is only available in clinical trials. ![]() Keeping in mind that gene therapy is only available for certain SCID types, what other ways can parents know if gene therapy is an appropriate treatment for their baby?Ī.
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