Gene therapy and retinitis pigmentosa: advances and future challenges
Gene therapy and retinitis pigmentosa: advances and future challenges
复制标题
DOI：
10.1002/bies.1092
复制
发表时间：
2001-07-01
期刊：
影响因子：
4
通讯作者：
Bennett, J
中科院分区：
文献类型：
--
作者：
Dejneka, NS;Bennett, J
有一天，也许可以使用基因疗法来治疗已发现遗传缺陷的疾病。由于许多与视网膜内遗传性眼部疾病有关的基因已被识别，因此眼部疾病是这种治疗形式的主要候选者。眼睛还具有易于接近、免疫学特性改变的优点，这是轻松传递病毒和避免全身免疫反应的重要考虑因素。目前，腺病毒、腺相关病毒和慢病毒已被成功用于将遗传物质转移至视网膜色素上皮和感光细胞。通过利用这些病毒的治疗基因，研究人员已经能够在啮齿动物视网膜色素变性模型中展示拯救作用，提供证据表明这种形式的疗法可以有效延迟感光细胞死亡。未来的挑战包括在眼睛在解剖学上与人类更相似的动物模型中确认治疗效果，并以最小的毒性证明长期救援。 BioEssays 23:662-668, 2001。(C) 2001 John Wiley & Sons, Inc.
It may be possible, one day, to use gene therapy to treat diseases whose genetic defects have been discerned. Because many genes responsible for inherited eye disorders within the retina have been identified, diseases of the eye are prime candidates for this form of therapy. The eye also has the advantage of being highly accessible with altered immunological properties, important considerations for easy delivery of virus and avoidance of systemic immune responses. Currently, adenovirus, adeno-associated virus and lentivirus have been used to successfully transfer genetic material to retinal pigment epithelium and photoreceptor cells. By harnessing therapeutic genes to these viruses, researchers have been able to demonstrate rescue in rodent models of retinitis pigmentosa, providing evidence that this form of therapy can be effective in delaying photoreceptor cell death. Future challenges include confirming therapeutic effects in animal models with eyes more anatomically similar to those of humans and demonstrating long-term rescue with minimal toxicity. BioEssays 23:662-668, 2001. (C) 2001 John Wiley & Sons, Inc.