The development of a novel gene therapy to treat age related macular degeneration (AMD)

“老年性黄斑变性(AMD)是一种影响视网膜中央部分(黄斑)的眼睛疾病，会使患者难以阅读、驾驶或识别人脸。湿性AMD(WAMD)可以迅速发展，在很短的时间内导致中心视力的严重变化--几天或几周，因为视网膜开始生长新的血管来试图解决这个问题。然而，这些新血管不会完全发育，反而会导致黄斑下的肿胀和出血，从而在患者的视野中心形成空白斑块。目前，英国约有60万人(主要是老年人)患有AMD导致的视力丧失，每年约有7万例新诊断病例。在全球范围内，wAMD的发病率约为1.7亿人。目前NHS对wAMD的治疗是使用一组名为抗血管内皮生长因子(anti-VEGF)抗体的药物，每隔4-6周直接向眼睛注射一次，以阻止新血管的生长和防止对黄斑的进一步损害。治疗方法需要频繁去眼科医生那里进行眼内注射，这令人不快，使患者面临感染的风险，可能导致角膜疤痕形成，随着时间的推移，视网膜/黄斑纤维化，这限制了与使用它们相关的长期改善。除了患者的负担，NHS的负担也很大，目前估计每年的成本约为16亿GB。这个项目的目的是开发一种新的wAMD基因疗法，可以通过一次注射安全地进行，从而实现长期、24小时稳定的治疗，预计将持续很多年甚至几十年，从而提高AMD患者的生活质量，同时显著降低NHS的成本。“

"Age-related macular degeneration (AMD) is an eye condition affecting the central part of the retina (the macula), which can make it difficult for patients to read, drive or recognise people's faces. The wet form of AMD (wAMD) can develop quickly, causing serious changes to central vision in a short period of time -- over days or weeks as the retina starts growing new blood vessels to try and address the problem. However, these new blood vessels do not fully develop and instead cause swelling and bleeding underneath the macular which leads blank patches in the centre of an individual's sight. Around 600,000 mainly elderly people in the UK currently have sight loss caused by AMD, with around 70,000 new cases diagnosed each year. Globally the incidence of wAMD affects around 170 million people.The current treatment for wAMD on the NHS is with a group of medicines called anti vascular endothelial growth factor (anti-VEGF) antibody-based drugs that are injected directly into the eye every 4-6 weeks to stop the growth of new blood vessels and preventing further damage to the macula.Treatment approaches require frequent visits to ophthalmic surgeons for intraocular injections, which are unpleasant, expose the patient to risk of infection, can cause corneal scarring and over time retinal/macular fibrosis, which limits the long-term improvements associated with their use. In addition to the burden on patients, the burden on the NHS is considerable, with costs currently estimated at around £1.6 billion annually.The aim of this project is to develop a new gene therapy for wAMD that can be safely administered with one single injection resulting in long-term, 24 hour stable treatment that would be expected to last for many years and even decades, thereby improving the quality of life for AMD patients while at the same time significantly reducing costs to the NHS."