微囊化基因工程干细胞移植技术在眼科疾病中的应用

Application of transplantation of microencapsulated engineered stem cells in ophthalmology

微囊化基因工程干细胞移植是指通过转染将目的基因导入靶细胞内，然后将该细胞微囊化并植入受体体内，从而达到既提高干细胞植入的组织相容性，且微囊内的功能细胞又可以长期存活并发挥其生物学效应，同时又可避免机体发生免疫排斥反应的目的的生物技术。该技术的发展使得异种组织细胞或基因工程细胞的移植成为可能。目前，微囊化基因工程干细胞移植已用于治疗视网膜色素变性（RP）的临床试验，取得了一定的疗效，并未发现严重并发症，为其他眼部慢性难治性疾病的治疗奠定了基础。就微囊化技术的原理，尤其是近年来基因工程干细胞在眼科疾病治疗中的应用进展进行综述，并展望微囊化基因工程干细胞移植技术在视网膜疾病中应用的可行性及前景。

Transplantation of microencapsulated genetic engineered stem cells is a new biological approach for the treatment of many refractory diseases. Transplantation of microencapsulated genetic engineered stem cells can transfect the interest gene into the target cells and further implants the gene to host after being microencapsulated. The microcapsules improve the survival time of target ceils and remain the ceils to play their biological function for a long- term duration, and the microencapsulated cells present with immunoisolation and good bioeompatibility after transplantation. This technology will make it possible for the xenotransplantation or gene engineered cell transplantation. Recently,transplantation of mieroencapsulated genetic engineered stem cells has been used in clinical trial of retinal pigment degeneration and made progress,which laid a foundation for the management of other chronic and refractory eye diseases. The concept and principle of the microeneapsulation technology are outlined, and the application of gene modification of stem ceils in ophthalmology are reviewed in this article.