Hansen John-Erik Stig, Gram Gregers J
Center for Små Handicapgrupper, og H:S Hvidovre Hospital, infektionsmedicinsk afdeling.
Ugeskr Laeger. 2002 Sep 9;164(37):4272-6.
The first positive clinical results of gene therapy trials have now become evident. The relatively few positive results and the numerous negative trials make it possible to identify both problems and potential for new development. The biggest problems have come from the viral vectors used for gene transfer. Most of the successful gene therapy trials have involved monogenetic diseases, where the relevant tissue has been isolated ex vivo, and where a retroviral vector has been inserted into the therapeutic gene in the nuclear DNA. Gene therapy seems to have a definite therapeutic potential in several rare, inherited diseases and also in certain acquired diseases, such as ischaemic heart disease.
基因治疗试验的首批积极临床结果现已显现。相对较少的积极结果和众多的阴性试验使得识别问题和新发展的潜力成为可能。最大的问题来自用于基因转移的病毒载体。大多数成功的基因治疗试验都涉及单基因疾病,其中相关组织已在体外分离,并且逆转录病毒载体已被插入核DNA中的治疗基因。基因治疗在几种罕见的遗传性疾病以及某些后天性疾病(如缺血性心脏病)中似乎具有明确的治疗潜力。