Banin Eyal, Chowers Itay
Department of Ophthalmology, Hadassah-Hebrew University Hospital and Medical School, Jerusalem.
Harefuah. 2004 May;143(5):368-71, 389.
Retinitis Pigmentosa (RP) is a heterogeneous group of inherited diseases that cause retinal degeneration, often leading to blindness. Over the last decade, significant insights have been obtained into the mechanisms underlying retinal degeneration in RP. This improved understanding of the pathogenesis of RP, combined with advances in molecular and cellular biology methods as well as electro-optical technologies, have contributed to renewed interest and increasing efforts to design novel therapeutic approaches for RP. In the first part of this review (Harefuah, April 2003), we summarized recent developments in the fields of gene therapy for RP, the use of neurotrophic growth factors, and attempts to alter disease progression by vitamins and nutritional modifications. In this second part of the review, retinal and retinal pigment epithelium transplantation, the potential use of stem cells to replace degenerating retinal cells and attempts to develop a retinal prosthesis using electro-optical devices are described. The exciting progress made in these various experimental directions raises hope that slowing or preventing the progression of retinal degeneration in RP patients, and perhaps even partial restoration of visual function in RP, is within reach.
视网膜色素变性(RP)是一组异质性遗传性疾病,可导致视网膜变性,常导致失明。在过去十年中,人们对RP视网膜变性的潜在机制有了重要认识。对RP发病机制的这种深入理解,再加上分子和细胞生物学方法以及电光技术的进步,促使人们重新关注并加大努力设计针对RP的新型治疗方法。在本综述的第一部分(《Harefuah》,2003年4月),我们总结了RP基因治疗领域、神经营养生长因子的应用以及通过维生素和营养调整改变疾病进展的尝试等方面的最新进展。在本综述的第二部分,将描述视网膜和视网膜色素上皮移植、利用干细胞替代退化视网膜细胞的潜在应用以及尝试使用电光设备开发视网膜假体的情况。这些不同实验方向所取得的令人振奋的进展让人看到希望,即减缓或阻止RP患者视网膜变性的进展,甚至可能部分恢复RP患者的视觉功能或许是可以实现的。