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Updated: May 5, 2026

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遺伝子療法:X-SCIDトランスゲン白血病原性

Adrian J Thrasher1, H Bobby Gaspar, Christopher Baum

  • 1Molecular Immunology Unit, Institute of Child Health, University College London, London WC1N 1EH, UK. a.thrasher@ich.ucl.ac.uk

Nature
|September 22, 2006
PubMed
まとめ

遺伝子療法は免疫機能を回復させることができますが,白血病のリスクがあります. この研究は,治療用遺伝子IL2RGが本質的に腫瘍性ではない可能性を示唆し,ヒト試験の安全性に関する懸念におけるその役割に疑問を投げかけています.

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Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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