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近年来,骨骼肌靶向基因转移主要有两条途径,一是间接体外(ex viro)基因修饰成肌细胞后将其植入骨胳肌组织;二是直接体内(in viro)骨骼肌内注射,原位转染成熟的非分裂肌细胞。间接体外实验周期长,临床适用性较差,但易获得较高水平和较长期的外源性基因的体内表达;直接体内法直接简便,接近临床应用的实际情况。本文对两条途径进行的基因免疫、基因治疗及外源性基因在骨骼肌中表达的调控等有关进展进行了一综述。
In recent years, there are mainly two ways to target gene transfer of skeletal muscle. One is to implant exoskeleton into skeletal muscle after indirect ex viro gene modification; the other is to directly inject intramuscular , In situ transfection of mature non-dividing muscle cells. Indirect long in vitro experiments, clinical applicability is poor, but easy to get higher levels and longer-term exogenous gene expression in vivo; direct in vivo method is simple and convenient, close to the actual situation of clinical application. This review summarizes the progress of gene immunization, gene therapy and the regulation of exogenous gene expression in skeletal muscle by two pathways.