医学
遗传增强
眼科
视神经病变
视神经
Leber遗传性视神经病
基因
生物
遗传学
作者
Patrick Yu‐Wai‐Man,Nancy J. Newman,Valério Carelli,Mark L. Moster,Valérie Biousse,Alfredo A. Sadun,Thomas Klopstock,Catherine Vignal,Robert C. Sergott,Günther Rudolph,Chiara La Morgia,Rustum Karanjia,Magali Taiel,Laure Blouin,Pierre Burguière,Gerard Smits,Caroline Chevalier,Harvey Masonson,Yordak Salermo,Barrett Katz
标识
DOI:10.1126/scitranslmed.aaz7423
摘要
At 96 weeks after unilateral intravitreal injection of rAAV2/2- ND4 , vision improved in both eyes in 78% of subjects affected with LHON.
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