Gene therapy of the corneal epithelium.
Gene therapy of the corneal epithelium.
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角膜上皮的基因治疗。
DOI：
10.1097/00004397-200404430-00010
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发表时间：
2004
影响因子：
--
通讯作者：
Azar,DimitriT
中科院分区：
文献类型：
--
作者：
Rosenblatt,MarkI;Azar,DimitriT
尽管有了很大的进步，传统的治疗方法已经不能充分地治疗许多涉及角膜上皮的常见疾病。诸如角膜新生血管、眼表结膜、反复糜烂、干眼综合征和神经营养性角膜病变等过程经常被发现对我们目前的治疗方式具有抵抗力。我们对导致这些疾病的分子机制的了解已经很多，但我们的治疗方法几乎没有反映出我们新获得的知识。我们的大多数上皮性疾病的治疗需要重复使用局部药物，这些药物并不是针对要治疗的疾病的病理生理学特定的，而是影响一系列生物过程，其中一个可能涉及到病理生理学。对于上述许多疾病过程，基因疗法形式的定制分子疗法可能被证明更有效，副作用更少(表1)。自从DNA被描述为基本的遗传密码以来，科学家和医生一直致力于利用我们对基因的理解来治疗人类疾病。基因疗法已经应用于许多疾病，在接下来的几年里，更多的基因疗法将成为我们临床医疗机构的一部分。1在利用基因打靶载体改变角膜中的基因表达作为潜在基因治疗的前奏方面，最近取得了进展。本文对基因载体进行了简要的讨论，并介绍了角膜上皮细胞基因转移的研究进展。将特别强调影响其作为基因治疗靶点的角膜上皮的特殊性质。
Despite much progress, conventional therapies have been unable to adequately treat many common disorders involving the corneal epithelium. Processes such as corneal neovascularization, conjuctivalization of the ocular surface, recurrent erosions, dry eye syndrome, and neurotrophic keratopathy are quite frequently found to be resistant to our current treatment modalities. Our knowledge of the molecular mechanisms responsible for these disorders has flourished, but few if any of our treatments reflect our newly acquired knowledge. Most of our treatment of epithelial disease requires the repeat usage of topical drugs that are not specific for the pathophysiology of the disease to be treated, but instead effect a host of biologic processes, one of which may be involved in the pathophysiology. For many of the aforementioned disease processes, tailored molecular therapy in the form of gene therapy may prove to be more effective and harbor fewer side effects (Table 1). Since the description of DNA as the fundamental genetic code, scientists and physicians have worked to use our understanding of genes to treat human disease. Gene therapy has been applied to a host of diseases, and over the next few years, more gene therapies will become part of our clinical armamentarium. 1 Recent progress has been made in the use of gene targeting vectors to alter gene expression in the cornea as a prelude to potential gene therapies. This review will briefly discuss gene vectors and describe the progress made in corneal epithelial gene transfer. Particular emphasis will be placed on special properties of the corneal epithelium that influence its potential as a target for gene therapy.