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Current Status of Gene Delivery and Gene Therapy in Lacrimal Gland using Viral Vectors

机译:病毒载体在泪腺中的基因传递和基因治疗的现状

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摘要

Gene delivery is one of the biggest challenges in the field of gene therapy. It involves the efficient transfer of transgenes into somatic cells for therapeutic purposes. A few major drawbacks in gene delivery include inefficient gene transfer and lack of sustained transgene expression. However, the classical method of using viral vectors for gene transfer has circumvented some of these issues. Several kinds of viruses, including retrovirus, adenovirus, adeno-associated virus, and herpes simplex virus, have been manipulated for use in gene transfer and gene therapy applications. The transfer of genetic material into lacrimal epithelial cells and tissues, both in vitro and in vivo, has been critical for the study of tear secretory mechanisms and autoimmunity of the lacrimal gland. These studies will help in the development of therapeutic interventions for autoimmune disorders such as Sjögren’s syndrome and dry eye syndromes which are associated with lacrimal dysfunction. These studies are also critical for future endeavors which utilize the lacrimal gland as a reservoir for the production of therapeutic factors which can be released in tears, providing treatment for diseases of the cornea and posterior segment. This review will discuss the developments related to gene delivery and gene therapy in the lacrimal gland using several viral vector systems.
机译:基因递送是基因治疗领域中的最大挑战之一。它涉及将转基因有效转移到体细胞中用于治疗目的。基因递送中的一些主要缺点包括基因转移效率低和缺乏持续的转基因表达。但是,使用病毒载体进行基因转移的经典方法已经解决了其中一些问题。已经操纵了几种病毒,包括逆转录病毒,腺病毒,腺伴随病毒和单纯疱疹病毒,用于基因转移和基因治疗应用。遗传物质在体外和体内转移至泪道上皮细胞和组织中,对于泪液分泌机制和泪腺自身免疫的研究至关重要。这些研究将有助于开发针对自身免疫性疾病的治疗性干预措施,例如与泪功能障碍有关的干燥综合征(Sjögren's syndrome)和干眼症(dry eye syndrome)。这些研究对于将来的努力也至关重要,这些努力利用泪腺作为产生可泪液释放的治疗因子的储库,为角膜和后段疾病提供治疗。这篇综述将讨论使用几种病毒载体系统在泪腺中与基因传递和基因治疗有关的发展。

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