美国联邦食品药品监督管理局批准的基因疗法恢复遗传失明. Penn Medicine team wins $3M prize for FDA-approved gene therapy restoring inherited blindness.

美国费城宾夕法尼亚大学医学院和儿童医院的一组研究人员获得了突破奖,因为他们开发出首个FDA批准的基因治疗遗传性失明. A team from Penn Medicine and Children’s Hospital of Philadelphia won the Breakthrough Prize for developing the first FDA-approved gene therapy for an inherited blindness. 他们的治疗方法,Luxturna, 可以恢复从小就失明的患者视力. Their treatment, Luxturna, restores sight to patients who would otherwise be blind from childhood. 这项研究启发了140多个针对视网膜疾病的基因疗法试验, 奖金为300万美元. The work has inspired over 140 gene therapy trials for retinal diseases and comes with a $3 million award.