Nayerossadat Nouri, Maedeh Talebi, Ali Palizban Abas
Molecular Genetic Laboratory, Alzahra Hospital, Isfahan University of Medical Sciences, Isfahan, Iran ; Pediatric Inherited Disease Research Center, Isfahan University of Medical Sciences, Isfahan, Iran.
Adv Biomed Res. 2012;1:27. doi: 10.4103/2277-9175.98152. Epub 2012 Jul 6.
Gene therapy is the process of introducing foreign genomic materials into host cells to elicit a therapeutic benefit. Although initially the main focus of gene therapy was on special genetic disorders, now diverse diseases with different patterns of inheritance and acquired diseases are targets of gene therapy. There are 2 major categories of gene therapy, including germline gene therapy and somatic gene therapy. Although germline gene therapy may have great potential, because it is currently ethically forbidden, it cannot be used; however, to date human gene therapy has been limited to somatic cells. Although numerous viral and nonviral gene delivery systems have been developed in the last 3 decades, no delivery system has been designed that can be applied in gene therapy of all kinds of cell types in vitro and in vivo with no limitation and side effects. In this review we explain about the history of gene therapy, all types of gene delivery systems for germline (nuclei, egg cells, embryonic stem cells, pronuclear, microinjection, sperm cells) and somatic cells by viral [retroviral, adenoviral, adeno association, helper-dependent adenoviral systems, hybrid adenoviral systems, herpes simplex, pox virus, lentivirus, Epstein-Barr virus)] and nonviral systems (physical: Naked DNA, DNA bombardant, electroporation, hydrodynamic, ultrasound, magnetofection) and (chemical: Cationic lipids, different cationic polymers, lipid polymers). In addition to the above-mentioned, advantages, disadvantages, and practical use of each system are discussed.
基因治疗是将外源基因组材料导入宿主细胞以产生治疗益处的过程。虽然最初基因治疗的主要重点是特殊的遗传疾病,但现在具有不同遗传模式的各种疾病和后天性疾病都成为了基因治疗的目标。基因治疗主要分为两大类,即生殖系基因治疗和体细胞基因治疗。虽然生殖系基因治疗可能具有巨大潜力,但由于目前在伦理上被禁止,所以不能使用;然而,迄今为止人类基因治疗仅限于体细胞。尽管在过去三十年中已经开发出了众多病毒和非病毒基因递送系统,但尚未设计出一种在体外和体内可无限制、无副作用地应用于所有类型细胞基因治疗的递送系统。在本综述中,我们将阐述基因治疗的历史、用于生殖系(细胞核、卵细胞、胚胎干细胞、原核、显微注射、精子细胞)和体细胞的所有类型的基因递送系统,包括病毒(逆转录病毒、腺病毒、腺相关病毒、辅助依赖型腺病毒系统、杂交腺病毒系统、单纯疱疹病毒、痘病毒、慢病毒、爱泼斯坦 - 巴尔病毒)和非病毒系统(物理方法:裸DNA、基因枪、电穿孔、流体动力学、超声、磁转染)以及(化学方法:阳离子脂质、不同的阳离子聚合物、脂质聚合物)。除上述内容外,还将讨论每个系统的优点、缺点和实际应用。