遗传性疾病的基因校正。

Fetal therapy Pub Date : 1989-01-01 DOI:10.1159/000263464
M P Johnson, A Drugan, O J Miller, M I Evans
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引用次数: 7

摘要

一些遗传性疾病可能是潜在的纠正通过引入和纳入正常基因到人体组织使用各种系统。尽管围绕整合、稳定表达和潜在的插入突变的技术问题尚未完全解决,但已经取得了足够的科学进展,从科学和伦理的角度来看,体细胞基因治疗都是可以接受的。组织特异性干细胞和胚胎干细胞移植将允许在胚胎发育早期进行治疗。随着技术问题的消除,这些程序将在道德上得到允许,因为它们将允许纠正毁灭性的遗传疾病。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Genetic correction of hereditary disease.

Several hereditary disorders may be potentially correctable by the introduction and incorporation of the normal gene into human tissues using a variety of systems. Although technical issues surrounding integration, stable expression and potential insertional mutagenesis to the treated cells has not yet been fully resolved, enough scientific progress has already been made to consider somatic cell gene therapy acceptable from both the scientific and ethical viewpoints. Tissue-specific stem cell and embryonic stem cell transplantation will allow therapy earlier in the developing embryo. As technical problems are eliminated, these procedures will become morally permissible, as they will allow the correction of devastating hereditary disease.

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