基因治療的困境：1996年的回顧評論

The Paradox of Gene Therapy -Review in 1996

周寬基(Kuan-Chih Chow) ； 陳光耀(Kuang Y. Chen)

《放射治療與腫瘤學》 3卷2期 (1996/06) Pp. 129-134

https://doi.org/10.6316/TRO/199603(2)129

摘要

基因治療雖然有其潛力，可是基因治療在科學和醫學上的能力仍然是非常有限。基因治療的人體試驗可分為遺傳性疾病的矯正與非遺傳性疾病的治療二大類，基因治療的第一個例子是穩性正染色體遺傳疾病中的先天性免疫缺失症，其可能病因是腺苷酸去胺酵素的缺失。傳統治療是定期注射放射線處理過的紅血球，或是聯結在聚乙烯甘醇上的酵素來緩解症狀，基因治療卻是以含有正常酵素基因的反轉錄病毒載體感染病人的T細胞後，再打回病人血中。病人則繼續接受傳統治療以強化療效。強化作用使基因治療的療效產生問題，究竟這些病人症狀的緩解，是否因為基因治療而得以改善，結論值得商榷。本文就最近基因治療的結果來討論基因治療必須考慮的幾個重要條件、目前使用的方法興可能的副作用，及一般民眾對基因治療的看法。

關鍵字

基因治療 ； 遺傳性疾病 ； 先天性免疫缺失症 ； 腺苷酸去胺酵素

並列摘要

Although gene therapy is potentially available, the accomplishment in scientific and medical application is still limited. Human clinical trial of gene therapy could be divided into two categories: correction of genetic diseases and treatment of non-genetic diseases. The first example of gene therapy is to treat patients with severe combined immunodeficiency, an autosomal recessive disease. The possible cause of the disease is the deficiency of adenosine deaminase. The traditional treatment is to inject the irradiated red blood cells or the polyethylene glycol-conjugated adenosine deaminase. The strategy of gene therapy is to transfect human T cells with retroviral vector that contains normal perspective gene and then to deliver these transfected human T cells back into the patient's circulation. Patient receives PEG-ADA continuously for the disease control. The belt-and-brace approach may be medically sensible. However, the call of success by gene therapy is in question. In this paper, we discuss several important issues of gene therapy, including the recent application methods and the plausible complications.

並列關鍵字

Gene therapy ； Genetic diseases ； Severe combined immunodeficiency ； Adenosine deaminase