Gaspar H B, Howe S, Thrasher A J
Molecular Immunology Unit, Institute of Child Health, London, UK.
Gene Ther. 2003 Nov;10(24):1999-2004. doi: 10.1038/sj.gt.3302150.
Severe combined immunodeficiencies have long been targeted as a group of disorders amenable to gene therapy because of their defined molecular biology and pathophysiology, and the prediction that corrected cells would have profound growth and survival advantage. Recently, several clinical studies have shown that conventional gene transfer technology can produce major beneficial therapeutic effects in these patients, but, as for all cellular and pharmacological treatment approaches, with a finite potential for toxicity.
由于严重联合免疫缺陷症具有明确的分子生物学和病理生理学特征，且预计校正后的细胞将具有显著的生长和生存优势，长期以来它一直被视为适合基因治疗的一组疾病。最近，几项临床研究表明，传统的基因转移技术可以在这些患者中产生重大的有益治疗效果，但是，与所有细胞和药物治疗方法一样，存在一定的潜在毒性。
