Advances in Gene Therapy for Diseases of the Eye.
Advances in Gene Therapy for Diseases of the Eye.
复制标题
DOI：
10.1089/hum.2016.040
复制
发表时间：
2016-08
影响因子：
4.2
通讯作者：
Punzo C
中科院分区：
文献类型：
--
作者：
Petit L;Khanna H;Punzo C
在过去的几年里，关于眼睛神经退行性疾病发病机制的分子机制的理解取得了巨大的进展。这些知识导致了基因治疗方法的发展，以治疗这些毁灭性的疾病。关于治疗性基因传递的疗效和效率的挑战推动了新的治疗方法的发展，这些方法继续发展眼科基因治疗领域。在这篇综述文章中，我们将讨论改善眼睛基因治疗的临床前和临床策略的发展，表明治疗视力丧失具有光明的前景。
Over the last few years, huge progress has been made with regard to the understanding of molecular mechanisms underlying the pathogenesis of neurodegenerative diseases of the eye. Such knowledge has led to the development of gene therapy approaches to treat these devastating disorders. Challenges regarding the efficacy and efficiency of therapeutic gene delivery have driven the development of novel therapeutic approaches, which continue to evolve the field of ocular gene therapy. In this review article, we will discuss the evolution of preclinical and clinical strategies that have improved gene therapy in the eye, showing that treatment of vision loss has a bright future.