GENE THERAPY--THE NEXT MILLENNIUM

基因治疗是分子医学的一种形式，它将在未来世纪对人类健康产生重大影响。 尽管有300项临床试验和6,000多名患者参加，但基因治疗尚未取得成功。这种失败的部分原因是缺乏适当的交付系统。 因此，会议旨在讨论改进的载体系统，其中将讨论各种不同的输送系统。 我们计划审查病毒载体和非病毒载体。 此外，会议的目的是提出这样的问题，即引入外源基因的免疫学后果是什么。 这将涉及细胞免疫反应和人体免疫反应。 会议还旨在询问最有用和最具信息量的动物模型系统是什么。此外，我们计划讨论如何调控基因的表达。 会议有几次会议，旨在讨论具体疾病以及正在考虑的治疗这些疾病的各种方法。 干细胞的研究也受到了相当大的重视，因为人们普遍认为，我们必须更多地了解干细胞的分化，以便了解基因如何被引入并持续表达一段时间。 总的来说，会议的重点是提出一个问题，即如何利用基础媒介学、免疫学和细胞生物学知识的改进来制造能够使外源基因持续表达的基因转移系统。 该会议是专为基因治疗的从业者以及对基因治疗领域感兴趣的学生而设计的。 会议也将迎合工业，因为许多基础科学的成功将不得不转移到那里，制药工业将是极其有价值的。 因此，本次会议的目的是能够将基础科学家、临床医生、学生和生物技术代表聚集在一起，使基因治疗成为现实，并成为常规的医学实践。

Gene therapy is a form of molecular medicine which will have a major impact on human health in the coming century. Although there are 300 clinical trials and over 6,000 patients enrolled, there has not yet been a successful outcome of gene therapy. Part of this failure can be attributed to the lack of an appropriate delivery system. Therefore the meeting is designed to discuss improved vector systems, where a wide variety of different delivery systems will be discussed. We plan to review both viral vectors and non-viral vectors. Additionally, the meeting is designed to ask the question as to what are the immunological consequences of introducing foreign genes. This will deal with both the cellular immune responses and the human immune responses. The meeting is also designed to ask what are the most useful and informative animal model systems. Additionally, we plan to discuss how the expression of the genes can be regulated. There are several sessions in the meeting which are designed to cover specific diseases as well as the various therapeutic modalities being considered for those diseases. There is also considerable emphasis on the study of stem cells because it is the general feeling that we have to know more about the stem cells which differentiate in order to understand how genes can be introduced and expressed for a sustained period of time. Overall the emphasis of the meeting is to ask the question of how improvements in basic vectorology, knowledge of immunology, and cell biology can be used to make systems of gene transfer which can give sustained expression of the foreign gene. The meeting is designed both for the practitioners of gene therapy as well as for students who are interested in the field of gene therapy. The meeting will also cater to industry because a lot of the successes of basic science will have to be transferred there, and the pharmaceutical industry will be extremely valuable. It is therefore the intention of this meeting to be able to bring together the basic scientists, the clinicians, the students and biotechnology representatives to be able to make gene therapy a reality as well as a routine practice of medicine.