基因转移治疗黄斑变性临床试验的积极结果
基因转移的长期安全性治疗新生血管年龄相关的黄斑变性(NVAMD),并在高级NVAMD患者眼中产生至少2.5岁的两种治疗蛋白的产生在结果发表了临床试验人类基因治疗。
在标题的文章中,“患有内皮抑素的慢病毒载体基因转移/血管抑制素(GEMU-LAR退化(GEM)研究,”Jeter Champochiadro,MD和威尔默眼科学院的MD和共陶道,Johns Hopkins Univery Medi-Cine(巴尔的摩,MD),Casey Eye Institute, Oregon Health & Science University (Portland, OR), University of Iowa Hospitals and Clinics (Iowa City), and Oxford BioMedica (Oxford, U.K.) present the design and results of the Phase 1 clinical trial. Each study participant re-ceived treatment in one eye consisting of a subretinal injection of a viral vector engineered to deliver two therapeutic genes, endostatin and angiostatin, each of which blocks the for-mation of blood vessels to combat the progression of NVAMD.
“这一直希望基因治疗对遗传视网膜疾病的壮观成功将转化为治疗更常见的眼睛的平台,”主编R. Flotte,MD,Celia和Isaac说Haidak医学Edu-Cation和Dean教授,Provost,以及Massachusetts Medical School大学,MA的Massachusetts Medical School大学的行政副校长。“Campochiadro博士对年龄相关的Macu-Lar变性(AMD)的开拓性工作展示了这种方法的可行性,这是一种最常见的视力威胁性疾病之一,其发病率急剧上升,因为我们的人口年龄较大。”
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