Douglas Joanne T
Division of Human Gene Therapy, Departments of Medicine, Pathology, and Surgery and the Gene Therapy Center, University of Alabama at Birmingham, Birmingham, AL, USA.
Methods Mol Biol. 2004;246:29-35. doi: 10.1385/1-59259-650-9:29.
Adenoviral vectors can be employed for gene delivery to skeletal muscle, both ex vivo and in vivo. Although the realization of the full potential of adenoviral vectors awaits the development of methods to allow safe and efficient targeted gene delivery to mature skeletal muscle upon intravenous vector administration (1), the current generation of vectors has nonetheless found utility in preclinical studies of gene therapy and in gene-transfer experiments designed to study muscle biology. Features of adenoviral vectors that have favored their use for gene delivery to skeletal muscle include the ability to infect both actively dividing and terminally differentiated cells, as well as their large insert capacity. Gutted adenoviral vectors are capable of carrying the large dystrophin gene together with regulatory sequences, and are therefore appropriate vehicles for gene-replacement therapy for Duchenne muscular dystrophy. In addition to their suitability for in vivo gene-therapy applications, adenoviral vectors have been used ex vivo to transfer genes to myoblasts prior to myoblast transplantation into muscle.
腺病毒载体可用于将基因递送至骨骼肌，包括体外和体内递送。尽管要充分发挥腺病毒载体的潜力，尚需开发出能够在静脉内给予载体后将基因安全、高效地靶向递送至成熟骨骼肌的方法（1），但目前一代的载体已在基因治疗的临床前研究以及旨在研究肌肉生物学的基因转移实验中得到应用。腺病毒载体有利于用于向骨骼肌进行基因递送的特性包括能够感染活跃分裂细胞和终末分化细胞，以及其较大的插入容量。去除了内部基因的腺病毒载体能够携带大型抗肌萎缩蛋白基因及其调控序列，因此是用于杜兴氏肌营养不良症基因替代疗法的合适载体。除了适用于体内基因治疗应用外，腺病毒载体还已在体外用于在将成肌细胞移植到肌肉之前将基因转移至成肌细胞。
