Gene therapy in corneal transplantation.

Gene therapy in corneal transplantation.
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DOI:
10.3109/08820538.2013.825297
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发表时间:
2013-09
影响因子:
1.7
通讯作者:
Hamrah P
Hamrah P
中科院分区:
医学4区
文献类型:
--
作者:
Qazi Y;Hamrah P

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角膜移植是最常进行的器官移植。角膜的免疫特权得到了广泛认可,部分原因是角膜移植的效果相对良好。在无血管、低风险环境中首次接受同种异体角膜移植的患者无需全身免疫抑制剂和组织相容性匹配即可获得 90% 的成功率。然而,免疫排斥仍然是移植失败的主要原因,特别是对于排斥风险高的患者。皮质类固醇仍然是预防和治疗免疫排斥的一线疗法。然而,目前的药理措施存在副作用大、重复使用、缺乏针对性、作用持续时间短等局限性。因此,实验性眼部基因治疗可能为免疫调节带来新的前景。从有效的病毒载体到可持续的选择性剪接,我们讨论了基因治疗在促进移植物存活方面的进展,并提出了基因介导的同种异体移植物排斥的预防的进一步途径。
Corneal transplantation is the most commonly performed organ transplantation. Immune privilege of the cornea is widely recognized, partly because of the relatively favorable outcome of corneal grafts. The first-time recipient of corneal allografts in an avascular, low-risk setting can expect a 90% success rate without systemic immunosuppressive agents and histocompatibility matching. However, immunologic rejection remains the major cause of graft failure, particularly in patients with a high risk for rejection. Corticosteroids remain the first-line therapy for the prevention and treatment of immune rejection. However, current pharmacological measures are limited in their side-effect profiles, repeated application, lack of targeted response, and short duration of action. Experimental ocular gene therapy may thus present new horizons in immunomodulation. From efficient viral vectors to sustainable alternative splicing, we discuss the progress of gene therapy in promoting graft survival and postulate further avenues for gene-mediated prevention of allogeneic graft rejection.
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