Adenovirus-mediated gene delivery to skeletal muscle.
Adenovirus-mediated gene delivery to skeletal muscle.
复制标题
腺病毒介导的基因递送至骨骼肌。
DOI：
10.1385/1-59259-650-9:29
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发表时间：
2004
影响因子：
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通讯作者：
J. T. Douglas
中科院分区：
文献类型：
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作者：
J. T. Douglas
腺病毒载体可用于离体和体内向骨骼肌的基因递送。尽管腺病毒载体的全部潜力的实现等待方法的开发，以允许在静脉内载体施用后安全和有效地将靶向基因递送到成熟骨骼肌（1），但是当前一代的载体已经在基因治疗的临床前研究和设计用于研究肌肉生物学的基因转移实验中发现了效用。腺病毒载体的特征有利于其用于将基因递送到骨骼肌，包括感染活跃分裂细胞和终末分化细胞的能力，以及它们的大插入容量。去肠腺病毒载体能够携带大的肌营养不良蛋白基因以及调节序列，因此是用于杜氏肌营养不良症的基因替代疗法的合适载体。除了它们适用于体内基因治疗应用之外，腺病毒载体已被用于在成肌细胞移植到肌肉中之前将基因离体转移到成肌细胞中。
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.