Zhang Xiang, Li Gen-lin
Beijing Ophthalmology & Visual Sciences Key Laboratory, Beijing Tongren Eye Center, Beijing Tongren Hospital, Capital Medical University, Beijing 100730, China.
Zhonghua Yan Ke Za Zhi. 2009 Sep;45(9):855-9.
Neurotrophic factors (NTFs) are pro-survival, pleiotrophic polypeptides which can play an important role in growth, differentiation and function maintenance of neurons. Many of them have been applied to treat various neurodegenerative diseases. Retinitis pigmentosa (RP), characterized by the impairment of photoreceptor-retinal pigment epithelium complex, is a retinal neurodegenerative disease with highly genetic heterogeneity. NTFs, one of the therapy approaches not targeting the mutation gene, have been proved to be effective in multiple types of animal models of retinal degeneration. Recent success in delivery of NTF genes by viral vectors or encapsulated cell technology (ECT) to degenerative retina provides an efficient administration approaches with great improvement in therapeutic effects. Further exploration for NTFs in the aspects of modulation of expression, location of receptors, signaling pathways, short- and long-term effects including side-effects will establish the foundation for clinical application of NTFs.
神经营养因子（NTFs）是一类具有促存活作用的多效性多肽，在神经元的生长、分化及功能维持中发挥重要作用。其中许多已被应用于治疗各种神经退行性疾病。视网膜色素变性（RP）是以光感受器 - 视网膜色素上皮复合体受损为特征的视网膜神经退行性疾病，具有高度的遗传异质性。神经营养因子作为一种不针对突变基因的治疗方法，已在多种视网膜变性动物模型中被证明有效。近期通过病毒载体或封装细胞技术（ECT）将神经营养因子基因递送至退变视网膜取得的成功，提供了一种有效的给药途径，使治疗效果有了很大改善。在神经营养因子的表达调控、受体定位、信号通路以及短期和长期效应（包括副作用）等方面的进一步探索，将为神经营养因子的临床应用奠定基础。
