Gene therapy in man and mice: adenosine deaminase deficiency, ornithine transcarbamylase deficiency, and Duchenne muscular dystrophy.
Gene therapy in man and mice: adenosine deaminase deficiency, ornithine transcarbamylase deficiency, and Duchenne muscular dystrophy.
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人和小鼠的基因治疗：腺苷脱氨酶缺乏症、鸟氨酸转氨甲酰酶缺乏症和杜氏肌营养不良症。
DOI：
10.1007/978-1-4615-7703-4_12
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发表时间：
1991
影响因子：
--
通讯作者：
Caskey,CT
中科院分区：
文献类型：
--
作者：
Grompe,M;Mitani,K;Lee,CC;Jones,SN;Caskey,CT
基因治疗被定义为将功能基因传递和表达到内源性缺乏该基因的患者（或动物）的体细胞组织中。根据自然组织表达模式和受疾病过程影响最大的部位，不同的解剖部位可能是基因转导的目标。迄今为止，逆转录病毒介导的基因转移到造血干细胞是最先进的基因治疗系统，在不久的将来最有可能在临床应用。然而，我们的实验室正在进行额外的努力，以开发将基因转导到肝脏、肠道、骨骼肌和心肌中的有效方法。
Gene therapy is defined as the delivery and expression of a functional gene into somatic tissues of patients (or animals) endogenously deficient in this gene. Depending on the natural tissue pattern of expression and the sites most affected by disease processes, different anatomic sites may be the targets for gene transduction. To date, retrovirus-mediated gene transfer into hemopoietic stem cells is the most advanced of gene therapy systems and the most promising in terms of clinical applications in the near future. There are, however, additional efforts under way in our laboratory to develop efficient means of transducing genes into the liver, intestine, skeletal and cardiac muscles.