Müller-Reible C R
Institut für Humangenetik Biozentrum, Würzburg.
Z Kardiol. 1994;83 Suppl 6:5-8.
The term gene therapy summarizes the attempts to correct inherited or acquired genetic defects by molecular genetic techniques. The manipulation of somatic cells differs in fundamental ethical aspects from that of germ cells. Some technical aspects of gene transfer, integration, and expression are discussed as well as the conditions set by the nature of the defect to be corrected. Adenosine deaminase deficiency is used as an example to demonstrate current approaches and their limitations.
基因治疗这一术语概括了通过分子遗传学技术纠正遗传性或后天性基因缺陷的种种尝试。体细胞的操作在基本伦理方面与生殖细胞的操作有所不同。本文讨论了基因转移、整合和表达的一些技术方面，以及由待纠正缺陷的性质所设定的条件。以腺苷脱氨酶缺乏症为例来说明当前的方法及其局限性。
