Petit Lolita, Khanna Hemant, Punzo Claudio
1 Department of Ophthalmology and Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.
2 Department of Neurobiology, University of Massachusetts Medical School , Worcester, Massachusetts.
Hum Gene Ther. 2016 Aug;27(8):563-79. doi: 10.1089/hum.2016.040. Epub 2016 Jun 13.
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.
在过去几年中，我们对眼部神经退行性疾病发病机制的分子机制的理解取得了巨大进展。这些知识推动了基因治疗方法的发展，以治疗这些破坏性疾病。治疗性基因递送的有效性和效率方面的挑战推动了新型治疗方法的发展，这不断推动着眼部基因治疗领域的发展。在这篇综述文章中，我们将讨论改善眼部基因治疗的临床前和临床策略的演变，表明视力丧失的治疗前景光明。
