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Gene therapy for age-related macular degeneration

机译：年龄相关黄斑变性的基因治疗

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Introduction: In neovascular age related macular degeneration (nAMD), gene therapy to chronically express anti-vascular endothelial growth factor (VEGF) proteins could ameliorate the treatment burden of chronic intravitreal therapy and improve limited visual outcomes associated with real world' undertreatment.Areas covered: In this review, the authors assess the evolution of gene therapy for AMD. Adeno-associated virus (AAV) vectors can transduce retinal pigment epithelium; one such early application was a phase I trial of AAV2-delivered pigment epithelium derived factor gene in advanced nAMD. Subsequently, gene therapy for AMD shifted to the investigation of soluble fms-like tyrosine kinase-1 (sFLT-1), an endogenously expressed VEGF inhibitor, binding and neutralizing VEGF-A. After some disappointing results, research has centered on novel vectors, including optimized AAV2, AAV8 and lentivirus, as well as genes encoding other anti-angiogenic proteins, including ranibizumab, aflibercept, angiostatin and endostatin. Also, gene therapy targeting the complement system is being investigated for geographic atrophy due to non-neovascular AMD.Expert opinion: The success of gene therapy for AMD will depend on the selection of the most appropriate therapeutic protein and its level of chronic expression. Future investigations will center on optimizing vector, promoter and delivery methods, and evaluating the risks of the chronic expression of anti-angiogenic or anti-complement proteins.
机译：介绍：在新血管时代相关的黄斑变性（NAMD），基因治疗以慢性表达抗血管内皮生长因子（VEGF）蛋白质可以改善慢性玻璃体治疗的治疗负担，并改善与现实世界患者有关的有限视觉结果。覆盖：在本次审查中，作者评估了AMD基因治疗的演变。腺相关病毒（AAV）载体可以脱节视网膜颜料上皮;一种这种早期申请是Aav2递送的颜料上皮衍生因子基因的I阶段I试验中的先进NAMD。随后，对AMD的基因治疗转移到可溶性FMS样酪氨酸激酶-1（SFLT-1）的研究，内源表达的VEGF抑制剂，结合和中和VEGF-A。经过一些令人失望的结果，研究以新的载体为中心，包括优化的AAV2，AAV8和慢病毒，以及编码其他抗血管生成蛋白的基因，包括Ranibizumab，AfliBercept，血管抑制素和内抑素。此外，由于非新生血管AMD，正在研究靶向补体系统的基因疗法。患者认为：AMD的基因治疗的成功将取决于选择最合适的治疗蛋白及其慢性表达水平。未来的调查将用于优化载体，推动者和递送方法，评估抗血管生成或抗补体蛋白的慢性表达的风险。

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