首页> 美国卫生研究院文献>Genes >The Foundation Fighting Blindness Plays an Essential and Expansive Role in Driving Genetic Research for Inherited Retinal Diseases
【2h】

The Foundation Fighting Blindness Plays an Essential and Expansive Role in Driving Genetic Research for Inherited Retinal Diseases

机译:基金会对抗失明症在推动遗传性视网膜疾病的遗传研究中起着至关重要的作用

代理获取
本网站仅为用户提供外文OA文献查询和代理获取服务,本网站没有原文。下单后我们将采用程序或人工为您竭诚获取高质量的原文,但由于OA文献来源多样且变更频繁,仍可能出现获取不到、文献不完整或与标题不符等情况,如果获取不到我们将提供退款服务。请知悉。

摘要

The Foundation Fighting Blindness leads a collaborative effort among patients and families, scientists, and the commercial sector to drive the development of preventions, treatments, and cures for inherited retinal diseases (IRDs). When the nonprofit was established in 1971, it sought the knowledge and insights of leaders in the retinal research field to guide its research funding decisions. While the Foundation’s early investments focused on gaining a better understanding of the genetic causes of IRDs, its portfolio of projects would come to include some of the most innovative approaches to saving and restoring vision, including gene replacement/augmentation therapies, gene editing, RNA modulation, optogenetics, and gene-based neuroprotection. In recent years, the Foundation invested in resources such as its patient registry, natural history studies, and genetic testing program to bolster clinical development and trials for emerging genetic therapies. Though the number of clinical trials for such therapies has surged over the last decade, the Foundation remains steadfast in its commitment to funding the initiatives that hold the most potential for eradicating the entire spectrum of IRDs.
机译:基金会抗盲基金会领导着患者,家庭,科学家和商业部门之间的合作,以推动对遗传性视网膜疾病(IRD)的预防,治疗和治疗的发展。当非营利组织于1971年成立时,它寻求视网膜研究领域领导者的知识和见识,以指导其研究经费的决策。尽管基金会的早期投资着重于更好地了解IRD的遗传原因,但其项目组合将包括一些最新颖的方法来保存和恢复视野,包括基因替代/增强疗法,基因编辑,RNA调节,光遗传学和基于基因的神经保护。近年来,基金会投资于诸如患者登记,自然史研究和基因检测计划等资源,以支持新兴遗传疗法的临床开发和试验。尽管在过去十年中,此类疗法的临床试验数量激增,但基金会仍然坚定地致力于资助那些具有最大潜力消除所有IRD的举措。

著录项

相似文献

  • 外文文献
  • 中文文献
  • 专利
代理获取

客服邮箱:kefu@zhangqiaokeyan.com

京公网安备:11010802029741号 ICP备案号:京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有
  • 客服微信

  • 服务号