胶质细胞源性神经营养因子干预视网膜色素变性给药方式的研究进展

Research progress on ophthalmic delivery of glial cell line-derived neurotrophic factor’s intervention in retinitis pigmentosa

视网膜色素变性(retinitis pigmentosa,RP)的治疗目前仍处于探索阶段。胶质细胞源性神经营养因子(glial cell line-derived neurotrophic factor,GDNF)是目前研究中重要的神经营养因子,但其传统给药方式生物利用度相对较低。近年来GDNF安全有效的给药方式成为研究热点,包括经病毒载体或非病毒载体的基因工程法释药技术、经聚合物释放系统释药技术、经细胞移植释药技术、小分子触发器诱导产生GDNF等。本文就GDNF干预RP的给药方式进行综述。

Treatment for retinitis pigmentosa(RP)is still in an exploratory period at present.Glial cell line-derived neurotrophic factor(GDNF)is an important neurotrophic factor,but the conventional ophthalmic delivery of GDNF results in a very low bioavailability,so the safe and effective delivery of GDNF has become the hotspot in recent years,including viral or non-viral mediated gene therapy,polymer-release systems,cell transplantation and small molecules to induce GDNF.This article gives a brief overview on related research progress of new ophthalmic delivery of GDNF intervention in RP.