Colella Pasqualina, Cotugno Gabriella, Auricchio Alberto
Telethon Institute of Genetics and Medicine (TIGEM), Via Pietro Castellino 111, 80131 Naples, Italy.
Trends Mol Med. 2009 Jan;15(1):23-31. doi: 10.1016/j.molmed.2008.11.003. Epub 2008 Dec 25.
As gene therapy begins to produce its first clinical successes, interest in ocular gene transfer has grown owing to the favorable safety and efficacy characteristics of the eye as a target organ for drug delivery. Important advances also include the availability of viral and non-viral vectors that are able to efficiently transduce various ocular cell types, the use of intraocular delivery routes and the development of transcriptional regulatory elements that allow sustained levels of gene transfer in small and large animal models after a single administration. Here, we review recent progress in the field of ocular gene therapy. The first experiments in humans with severe inherited forms of blindness seem to confirm the good safety and efficacy profiles observed in animal models and suggest that gene transfer has the potential to become a valuable therapeutic strategy for otherwise untreatable blinding diseases.
随着基因治疗开始取得首批临床成功，由于眼睛作为药物递送靶器官具有良好的安全性和有效性特征，人们对眼部基因转移的兴趣与日俱增。重要进展还包括有能够有效转导各种眼细胞类型的病毒和非病毒载体，眼内给药途径的应用，以及转录调控元件的开发，这些元件可使在大小动物模型单次给药后实现持续水平的基因转移。在此，我们综述眼部基因治疗领域的最新进展。首批针对严重遗传性失明形式的人体实验似乎证实了在动物模型中观察到的良好安全性和有效性概况，并表明基因转移有可能成为治疗其他无法治愈的致盲疾病的有价值治疗策略。
