Gene therapy: regulatory issues and international approaches to regulation.
Gene therapy: regulatory issues and international approaches to regulation.
复制标题
基因治疗：监管问题和国际监管方法。
DOI：
10.1016/s0958-1669(97)80018-3
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发表时间：
1997
影响因子：
7.7
通讯作者：
O. Cohen
中科院分区：
文献类型：
--
作者：
O. Cohen
基因治疗的潜在应用极其广泛，从单基因遗传性疾病如杜氏肌营养不良症、溶酶体贮积病或囊性纤维化延伸到获得性疾病如艾滋病、癌症、血栓栓塞性心血管疾病和神经系统疾病。目前正在研究治疗性基因转移的潜力，以解决这样一个大面板的条件。基因疗法在未来肯定会成为一种主要的治疗选择，特别适用于没有可用替代治疗的疾病。重组DNA技术的发展引起了公众的恐惧和对潜在风险的猜测，事实上，基因治疗的实施涉及可能不会没有潜在副作用的技术方法（与许多解决严重疾病的传统治疗方法一样）。基因治疗的监管旨在评估潜在的风险，以限制它们，但也要划定一个安全的边界。这应有助于更好地理解和提高公众的接受程度。
Potential applications of gene therapy are extremely numerous, extending from monogenic hereditary disorders such as Duchenne muscular dystrophy, lysosomal storage diseases or cystic fibrosis to acquired disorders like AIDS, cancer, thrombo-embolic cardiovascular diseases and neurological disorders. The potential of therapeutic gene transfer addressing such a large panel of conditions is currently being investigated. Gene therapy will certainly represent, in the future, a major therapeutic option applying in particular to disorders with no available alternative treatment. Development of recombinant DNA technology has induced public fears and speculation regarding potential risks and in fact, implementation of gene therapy involves technological approaches that might not be devoid of potential side effects (as with many conventional therapeutic means of addressing severe conditions). Regulation of gene therapy is intended to assess potential risks in order to restrict them, but also to delineate a margin where safety is being secured. This should contribute to better understanding and improved public acceptance.