Prospects for gene therapy in corneal disease
Prospects for gene therapy in corneal disease
复制标题
DOI：
10.1038/sj.eye.6700565
复制
发表时间：
2003-11-01
期刊：
影响因子：
3.9
通讯作者：
Larkin, DFP
中科院分区：
文献类型：
--
作者：
Jun, AS;Larkin, DFP
已经使用病毒和非病毒载体将cDNA转移至角膜细胞。与其他组织一样，使用组织化学或荧光标记基因进行了研究，检查载体介导的基因转移到角膜的可行性和最佳方法。这些方法在体外使用角膜细胞或细胞系，并将整个角膜维持在离体培养物中。已经使用实验模型对特定角膜疾病（例如同种异体移植排斥、准分子激光疤痕和单纯疱疹性角膜炎）进行了基于基因的干预措施的检验。随着角膜细胞基因改造的可行性已被成功证明，基因治疗载体在人类角膜疾病的治疗中具有巨大的潜力。基因转移载体的持续改进将提高基因治疗的功效和安全性。除了使用 cDNA 转移作为获得性角膜疾病的药物或蛋白质治疗的替代方法之外，我们对遗传性角膜疾病的遗传基础的不断了解将最终导致针对此类疾病开发出特异性且有效的基因疗法。
Transfer of cDNA to corneal cells has been accomplished using viral and nonviral vectors. Studies examining the feasibility and optimal methods for vector-mediated gene transfer to the cornea have, as in other tissues, been performed using histochemical or fluorescent marker genes. These have used corneal cells or cell lines in vitro, and whole corneas maintained in ex vivo culture. Gene-based interventions have been examined in specific corneal disorders such as allograft rejection, postexcimer laser scarring, and herpes simplex keratitis using experimental models.As the feasibility of genetic modification of corneal cells has been successfully demonstrated, there is great potential for gene therapy vectors in the treatment of human corneal disease. Continued improvements in vectors for gene transfer will improve the efficacy and safety of gene therapy. In addition to use of cDNA transfer as an alternative to drug or protein treatments in acquired corneal disorders, our expanding knowledge of the genetic basis of inherited corneal disorders will ultimately lead to the development of specific and effective gene therapies in this category of diseases.