Phase I gene therapy trial in Israeli patients with Leber Congenital Amaurosis caused by a founder RPE65 mutation: safety and efficacy update with up to three years of follow-up

被引:0
作者
Banin, E. [1 ]
Obolensky, A. [1 ]
Hemo, Y. [1 ]
Marks-Ohana, D. [1 ]
Sela, M. [1 ]
Yamin, E. [1 ]
Hauswirth, W. W. [2 ]
Jacobson, S. [3 ]
Sharon, D. [1 ]
机构
[1] Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, Israel
[2] Univ Florida, Gainesville, FL 32611 USA
[3] Univ Penn, Scheie Eye Inst, Philadelphia, PA 19104 USA
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中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
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页码:A6 / A7
页数:2
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