Age-related macular degeneration treatment in the era of molecular medicine

作     者：Rick N Nordgren Ahmed M Elkeeb Bernard F Godley 

作者机构：Department of Ophthalmology and Visual SciencesUniversity of Texas Medical BranchGalvestonTX 77555United States 

出 版 物：《World Journal of Ophthalmology》 (世界眼科杂志)

年 卷 期：2014年第4卷第4期

页      面：130-139页

学科分类：1002[医学-临床医学] 100212[医学-眼科学] 10[医学] 

主　　题：Age-related macular degeneration Stem cell therapy Anti-vascular endothelial growth factor Gene therapy Complement factor H High-temperature requirement factor A1 Age-relateed maculopathy susceptibility 2 Pharmacogenomics Genetics 

摘      要：Age-related macular degeneration(AMD) is the leading cause of irreversible blindness in the developed world. The quality of life of both patients and families is impacted by this prevalent disease. Previously, macular degeneration had no known effective treatment. Today, vitamins for non-exudative AMD and intravitreal injection of medications for its exudative form are primary forms of current treatment. Modern advances in molecular science give rise to new possibilities of disease management. In the year 2003 the sequencing of the entire human genome was completed. Since that time, genes such as complement factor H, high-temperature requirement factor A1, and age-relateed maculopathy susceptibility 2 have been discovered and associated with a higher risk of AMD. A patient s genetic make-up may dictate the effectiveness of current or future therapeutic options. In addition, utilizing genetic data and incorporating it into new treatments(such as viral vectors) may lead to longer-lasting(or permanent) VEGF blockade and specific targeting of complement related genes. There have also been considerable advances in stem cell directed treatment of AMD. Retinal pigment epithelial(RPE) cells can be derived from human embryonic stem cells, induced pluripotent stem cells, or adult human RPE stem cells. Utilizing animal models of RPE and retinal degeneration, stem cell-derived RPE cells have been successfully implanted into the subretinal space. They have been injected as a cell mass or as a pre-prepared monolayer on a thin membrane. Visual recovery has been demonstrated in a retinal dystrophic rat model. Preliminary data on 2 human subjects also demonstrates possible early visual benefit from transplantation of stem cell-derived RPE. As more data is published, and as differentiation and implantation techniques are optimized, the stabilization and possible improvement of vision in individuals with non-exudative macular becomes a real possibility. We conclude that the technologi