这是摘自抗盲基金会刚发给我的一则消息，应该算是个好消息，虽然还比较遥远。也试着稍加翻译修改，和大家分享一下。
An international research team, funded in part by the Foundation Fighting Blindness, has used gene therapy to reactivate retinal cells that were previously unresponsive to light. The treatment was delivered to cones, the retinal cells that provide central and daytime vision. Investigators evaluated the gene therapy in two mouse models of retinal degenerative disease, as well as cultures of human retinal tissue. Using a variety of tests, the researchers demonstrated that the treated cells restored functional vision, and that the restoration of vision persisted over a long period of time.
In retinal degenerative diseases such as retinitis pigmentosa, cones stop providing vision before they die and can remain in a compromised state for several years. The gene therapy used to restore vision in the cone-reactivation study leads to the production of a light-sensitive protein called halorhodopsin, which is similar to vision-activating proteins occurring naturally in the retina. The presence of halorhodopsin appears to reactivate the visual cycle, the biochemical process in retinal cells that lead to vision.
An adeno-associated virus (AAV), a therapeutic virus, was used to deliver the treatment to the cones. AAVs are the same delivery mechanism used in landmark gene therapy clinical trials that have restored vision in 28 children and young adults who were virtually blind from Leber congenital amaurosis.
Dr. José-Alain Sahel, one of the cone-reactivation study investigators and director of the Foundation-funded Paris Research Center for the Study of Retina Degenerative Diseases, says that through high-resolution imaging, his team has identified people who may be good candidates for a future clinical trial. He adds that even people who are legally blind might have some cones reactivated and vision restored.
Dr. Sahel also notes that the cone-reactivation treatment has the potential to be even more beneficial if coupled with a therapy that helps protect cones from degeneration. Dr. Sahel and his team are planning a clinical trial of a neuroprotective treatment called rod-derived cone viability factor, or RdCVF, which has shown excellent results for preserving cones in preclinical studies.
The cone-reactivation study was led by Dr. Botond Roska of the Friedrich Miescher Institute for Biomedical Research in Switzerland, and included scientists from several European research institutions. Results of the study were published in the June 24 issue of the journal Science (online edition).
一个国际研究小组，在部分资金由抗盲基金会提供，已经利用基因疗法来恢复了以前没有反应视网膜细胞。治疗视锥细胞，即中央和提供白天视野视网膜细胞。调查评估了在两个视网膜退行性疾病小鼠模型的基因治疗，以及人体视网膜组织的人文研究。使用各种测试，研究人员表明，经处理后的细胞恢复功能视力，而且视力恢复较长期坚持。
在视网膜变性疾病，如色素性视网膜炎，视锥体停止提供视力死亡之前，可以在妥协的状态持续数年。该基因疗法用于恢复锥体恢复研究的视野导致了一个光敏感蛋白生产所谓halorhodopsin（盐细菌视紫红质） ，它类似于激活视网膜蛋白质自然产生。在场的halorhodopsin似乎恢复了视觉周期，导致视力的视网膜细胞的生化过程。
一个腺病毒群（ AAV）病毒治疗物质，是用于运送治疗物到视锥细胞。自动增值服务是相同的传送机制，该具有里程碑意义的基因治疗已经恢复了28个孩子即莱伯先天性黑朦失明的年轻人视野的临床试验。
何博士，阿兰萨赫勒，锥，重新调查和研究基金会资助的巴黎研究中心的视网膜退行性疾病的研究主任之一，他表示，通过高分辨率成像，他的团队已经确定的人谁可能是好的候选人为今后的临床试验。他还说，即使在法律上的人谁可能有一些盲人和视力恢复锥恢复。
萨赫勒博士还指出，锥恢复治疗，有可能会更加有利，如果有一种疗法，可以帮助保护细胞免继续变性。萨赫勒博士和他的团队正在计划一项名为神经保护杆源性锥活力因子，或RdCVF ，这表明为维护良好的临床前研究结果锥治疗的临床试验。
该锥恢复研究由博士博通德罗斯卡的弗里德里希米歇尔生物医学研究所在瑞士，其中包括来自欧洲几个研究机构的科学家。这项研究结果发表在6月24日刊 科学 （在线版） |
