Gene therapy for sickle cell disease.
對於鐮刀型細胞的基因治療法
歐羅萵A, 歐可文杜 CI
拉哥斯(奈及利亞的首都)大學教學醫院, 奈及利亞拉哥斯的小艇碼頭郵局信箱8893號
醫學名詞：
1.Sickle cell disease 鐮刀型細胞疾病
2.genetic 遺傳
3.disorders 症狀.疾病
4.hemoglobin 血紅素
5.allele 對偶基因
6.polymerization 聚合作用
7.Autosomal 體染色體
8.recessive 隱性
9.gene therapy 基因治療
10.diseased cells 有害的細胞
11.mutant gene 基因突變
2.genetic 遺傳
3.disorders 症狀.疾病
4.hemoglobin 血紅素
5.allele 對偶基因
6.polymerization 聚合作用
7.Autosomal 體染色體
8.recessive 隱性
9.gene therapy 基因治療
10.diseased cells 有害的細胞
11.mutant gene 基因突變
Abstract
摘要
BACKGROUND: Sickle cell disease encompasses a group of genetic disorders characterized by the presence of at least one hemoglobin S (Hb S) allele,
背景：鐮刀型細胞疾病包含一組具有至少一個血紅素(血色素)對偶基因存在特徵的混亂基因
and a second abnormal allele that could allow abnormal hemoglobin polymerisation leading to a symptomatic disorder.
而且第二個不正常的對偶基因會讓不正常的血紅素聚合作用而導致混亂的症狀，
Autosomal recessive disorders (such as sickle cell disease) are good candidates for gene therapy because a normal phenotype can be restored in diseased cells with only a single normal copy of the mutant gene.
因為一個正常顯型細胞可以藉著只有一個單獨、正常的突變基因複製品的不健全細胞得以恢復。
因為一個正常顯型細胞可以藉著只有一個單獨、正常的突變基因複製品的不健全細胞得以恢復。
OBJECTIVES: The objectives of this review are:- to determine whether gene therapy can improve survival and prevent symptoms and complications associated with sickle cell disease;
目標：本次審查的目標是：確定基因療法是否可以提高生存率，防止鐮狀細胞疾病的併發症；
- to examine the risks of gene therapy against the potential long-term gain for people with sickle cell disease.
針對潛在長期鐮狀細胞疾病的人研究基因治療的風險與益處。
SEARCH STRATEGY: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Haemoglobinopathies Trials Register,
搜索策略：我們檢索了柯克倫纖維性囊腫和遺傳性聚合型紅血球蛋白病試驗紀錄，
which comprises of references identified from comprehensive electronic database searches and searching relevant journals and abstract books of conference proceedings.
其中包括引用正確的全方位電子資料庫檢索和搜索有關的期刊和書籍摘要的會刊。
Date of the most recent search of the Group's Haemoglobinopathies Trials Register: 05 March 2010.
最近搜索聚合型紅血球蛋白病試驗紀錄的日期：2010年3月5日 。
SELECTION CRITERIA: All randomised or quasi-randomised clinical trials (including any relevant phase 1, 2 or 3 trials) of gene therapy for all individuals with sickle cell disease, regardless of age or setting.
選擇標準：所有隨機或半隨機臨床試驗(包括任何有第一期，2或3期試驗)基因療法所有個人鎌狀細胞疾病，不論年齡或設定。
DATA COLLECTION AND ANALYSIS: No trials of gene therapy for sickle cell disease were found.
數據收集和分析：沒有試驗被發現於基因治療鎌狀細胞病。
MAIN RESULTS: No trials of gene therapy for sickle cell disease were reported.
MAIN RESULTS: No trials of gene therapy for sickle cell disease were reported.
主要結果：沒有試驗被發現於基因治療鎌狀細胞疾病。
AUTHORS' CONCLUSIONS: No randomised or quasi-randomised clinical trials of gene therapy for sickle cell disease were reported.
作者結論：沒有隨機或半隨機臨床試驗被發現為基因治療於鎌狀細胞疾病。
Thus, no objective conclusions or recommendations in practice can be made on gene therapy for sickle cell disease. 因此在實驗上基因療法治療鎌狀細胞疾病，沒有客觀的結論或建議。
This systematic review has identified the need for well-designed, randomised controlled trials to assess the benefits and risks of gene therapy for sickle cell disease. 關於基因療法對於鎌狀細胞疾病這個系統回顧需要精心設計隨機對照試驗以評估的好處和風險。
