• 主题
高级搜索  >
历史搜索 清空历史
首页> 外文期刊>Expert opinion on biological therapy >AAV-mediated gene transfer for retinal diseases.
【24h】

AAV-mediated gene transfer for retinal diseases.

机译：AAV介导的视网膜疾病基因转移。

获取原文
获取原文并翻译 | 示例
           
页面导航
  • 摘要
  • 著录项
  • 相似文献
  • 相关主题

摘要

Vectors based on the adeno-associated virus (rAAV) are able to transduce the retina of animal models, including non-human primates, for a long-term period, safely and at sustained levels. The ability of the various rAAV serotypes to transduce retinal target cells has been exploited to successfully transfer genes to photoreceptors, retinal pigment epithelium and the inner retina, which are affected in many inherited and non-inherited blinding diseases. rAAV-mediated, constitutive and regulated gene expression at therapeutic levels has been achieved in the retina of animal models, thus providing proof-of-principle of gene therapy efficacy and safety in models of dominant and recessive retinal disorders. In addition, gene transfer of molecules with either neurotrophic or antiangiogenic properties provides useful alternatives to the classic gene replacement for treatment of both mendelian and complex traits affecting the retina. Years of successful rAAV-mediated gene transfer to the retina have resulted inrestoration of vision in dogs affected with congenital blindness. This has paved the way to the first attempts at treating inherited retinal diseases in humans with rAAV. Although the results of rAAV clinical trials for non-retinal diseases give a warning that the outcome of viral-mediated gene transfer in humans may be different from that predicted based on results in other species, the immune privilege of the retina combined with the versatility of rAAV serotypes may ultimately provide the first successful treatment of human inherited diseases using rAAV.
机译：基于腺相关病毒（rAAV）的载体能够长期安全，持续地转导动物模型的视网膜，包括非人类灵长类动物。已经利用各种rAAV血清型转导视网膜靶细胞的能力来成功地将基因转移到感光细胞，视网膜色素上皮和内部视网膜，这些基因在许多遗传性和非遗传性致盲疾病中都受到影响。在动物模型的视网膜中已经实现了rAAV介导的，组成型和调节型基因水平的治疗，从而在显性和隐性视网膜疾病模型中提供了基因治疗功效和安全性的原理证明。此外，具有神经营养或抗血管生成特性的分子的基因转移为经典的基因替代提供了有用的替代方法，可用于治疗影响视网膜的孟德尔和复杂性状。多年成功地通过rAAV介导的基因转移到视网膜已导致先天性失明的犬的视力恢复。这为rAAV治疗人类遗传性视网膜疾病的首次尝试铺平了道路。尽管针对非视网膜疾病的rAAV临床试验的结果警告说，病毒介导的人类基因转移的结果可能与其他物种的结果不同，但视网膜的免疫特权与人类的多功能性相结合。 rAAV血清型最终可能会使用rAAV首次成功治疗人类遗传性疾病。

著录项

相似文献

  • 外文文献
  • 中文文献
  • 专利
获取原文

客服邮箱：kefu@zhangqiaokeyan.com

京公网安备：11010802029741号 ICP备案号：京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有

  • 客服微信

  • 服务号