What Is Next for Retinal Gene Therapy?
What Is Next for Retinal Gene Therapy?
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DOI：
10.1101/cshperspect.a017442
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发表时间：
2015-04-15
影响因子：
5.4
通讯作者：
Vandenberghe LH
中科院分区：
文献类型：
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作者：
Vandenberghe LH
视网膜致盲疾病的基因治疗领域正在经历令人难以置信的势头，这是由遗传性视网膜变性的早期临床试验的希望结果所证明的。将该基因用作药物的前提已经走过了很长的路，并且可能已经在视网膜疾病的治疗中找到了它的利基。事实上，由于视网膜适应症的治疗选择有限，基因治疗已被证明是可行的，安全的，有效的，并可能导致单次注射后的持久效果。在这里，我们的目的是把背景下的承诺和潜力，技术，临床和经济的界限限制其应用和发展，并推测未来的基因治疗是一个不可分割的组成部分，眼科临床护理。
The field of gene therapy for retinal blinding disorders is experiencing incredible momentum, justified by hopeful results in early stage clinical trials for inherited retinal degenerations. The premise of the use of the gene as a drug has come a long way, and may have found its niche in the treatment of retinal disease. Indeed, with only limited treatment options available for retinal indications, gene therapy has been proven feasible, safe, and effective and may lead to durable effects following a single injection. Here, we aim at putting into context the promise and potential, the technical, clinical, and economic boundaries limiting its application and development, and speculate on a future in which gene therapy is an integral component of ophthalmic clinical care.