神经营养因子治疗视网膜色素变性的研究进展

神经营养因子是能够促进神经元存活、生长、分化及维持其功能的多效性肽类因子的总称,可被作为有效的神经保护剂用于治疗多种神经变性类疾病.视网膜色素变性(RP)是以光感受器-视网膜色素上皮复合体损害为主的高度遗传异质性视网膜变性疾病,神经营养因子作为不针对致病基因的RP治疗策略,其疗效已在多种视网膜变性的动物模型中得到证实.以病毒为载体的转基因治疗和细胞包囊技术为神经营养因子提供了有效的给药途径,可使疗效明显提高.对神经营养因子在视网膜中的表达及其调节、受体分布特点、作用通路、疗效及副作用等方面的深入研究为神经营养因子的临床应用奠定了基础.

Progress in treatment of retinitis pigmentosa by neurotrophic factors

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.