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引言
基因治疗是指改变细胞遗传物质为基础的医学治疗。目前仅限于体细胞。基因治疗的技术和方式日趋多样性。按基因导入的形式，分为体外基因导入（exvivo）及体内基因导入（invivo）两种形式。前者是在体外将基因导入人细胞，然后将该细胞注入人体。其制品形式是外源基因转化的细胞，适合在具有专门技术人才和GMP条件的医疗单位进行。后者则是将基因通过适当的导入系统直接导入人体，包括病毒的与非病毒的方法。其制品形式是基因工程技术改造的病毒或者是重组DNA、或者是DNA复（混）合物。基因治疗制剂种类较多，因此，本指导原则不可能用一个模式来概括，只能提出一个共同的原则，具体的方案应根据这些原则，确定研究技术路线。其基本原则：一是必须确保安全与有效，要充分估计可能遇到的风险，并提出相应的质控要求；二是要促进基因治疗的研究，并加强创新。对一些新的治疗技术路线的相应质控要求，可有一定的灵活性，应注意到基因治疗本身的特点以及它与经典的化学合成药物或基因工程药物的差别。目前，一些基因治疗研究相对比较成熟，而一些则不够完善，更加要求研究者在使用该技术指导原则时不可生搬硬套。为此，研究者应加强咨询和论证，提出一个科学可行的研究方案，最终获得确保安全有效的基因治疗制品。
Introduction
Gene therapy refers to the medical therapy based on change of the genetic materials of cells. For the moment, it is only limited to somatic cells. In recent years, the techniques and methods of gene therapy have been more and more various. According to the types of gene transfer, it can be divided into ex vivo gene transfer and in vivo gene transfer. The former refers to ex vivo transfer of genes into human cells and infusion of cells into human body. Its preparations are the cells transformed with extraneous genes and are suitable for use in the medical institutions with professional staff conforming to the regulations of GMP. The latter refers to direct introduction of genes into human body with appropriate introduction system, including viral and non-viral methods. Its preparations are viruses modified with gene engineering techniques or recombinant DNA or the compounds (mixtures) of DNA. Gene therapy has many types; therefore, this guideline cannot be summarized as one pattern, only a common principle can be put forward, and as for specific protocol, the technical route of research should be determined based on these principles. The two cardinal principles are as follows: first, the safety and effectiveness should be ensured, the potential risks should be sufficiently estimated, and the relevant quality control requirements should be put forward; second, the researches on gene therapy should be facilitated, and innovation should be intensified. The quality control requirements for some new therapeutic technical routes are flexible. The characteristics of gene therapy and its difference from classical chemically synthetic drugs or gene engineering drugs should be paid attention to. For the moment, some gene therapy researches are comparatively mature, while some are imperfect, so the investigator cannot apply the technical guideline mechanically. For this reason, investigator should enhance the consultation and demonstration, put forward a scientific and feasible study protocol, and finally obtain a safe and effective gene therapy preparation.
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