Gene/Protein Disease Symptom Drug Enzyme Compound
Pivot Concepts:   Target Concepts:
Query: UMLS:C0021051 (immunodeficiency)
 71,517 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Three human fetuses were treated by transplantation of human fetal liver stem cells. Two of them had severe immunodeficiency disease and the third had thalassaemia major. All three in-utero transplants were followed by engraftment. No adverse effect was seen. The three patients have now been born: the first is very healthy thanks to the reconstitution of cell-mediated immunity associated with this transplant, and he lives normally at home; the other two, who were treated more recently, have not yet shown a complete effect. The feasibility and efficacy of this procedure, used for the first time in humans, has therefore been demonstrated: during early fetal development, foreign cells engraft readily and may result in significant improvement of a large variety of inherited diseases.
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PMID:In-utero transplantation of fetal liver stem cells into human fetuses. 155 56

Three human fetuses were treated by stem cell transplantation from fetal liver. Two of them had severe immunodeficiency disease and the third one had thalassemia major. All three in utero transplants were followed by engraftment. No adverse effect was seen. The three patients are now born: the first one is cured, the two other ones are still under investigation. This procedure, for the first time used in humans, is therefore shown to be an effective and safe therapy of inherited diseases, during early fetal development.
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PMID:In utero transplantation of stem cells in humans. 198 27

We report the case of an infant with severe combined immunodeficiency who was presented with PIV3 infection. Aerosolized ribavirin was administered for 10 months until the child gained a functional immune system through an allogeneic hematopoietic stem cell transplant and cleared PIV3 infection. No adverse effect was observed in the child and in healthcare personnel, with a follow-up of three years. Despite the burden of aerosolized administration, early and prolonged administration of aerosolized ribavirin was feasible, well tolerated, and safe for the patient and the caregivers. This is a case report and no definite conclusions can be drawn. However, our experience suggests that prolonged aerosolized ribavirin administration should be considered for the treatment of PIV3 infection in the context of primary immunodeficiency, where there is no currently available alternative treatment, until a functional immune system is gained.
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PMID:Long-term therapy with aerosolized ribavirin for parainfluenza 3 virus respiratory tract infection in an infant with severe combined immunodeficiency. 1730 Apr 87