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Seven-Year Follow-up of Gene Therapy for Leber's Hereditary Optic Neuropathy.
Ophthalmology ( IF 13.1 ) Pub Date : 2020-02-25 , DOI: 10.1016/j.ophtha.2020.02.023
Jiajia Yuan 1 , Yong Zhang 2 , Hongli Liu 1 , Dan Wang 1 , Yangyang Du 1 , Zhen Tian 2 , Xin Li 2 , Shuo Yang 3 , Han Pei 4 , Xing Wan 4 , Su Xiao 5 , Lin Song 1 , Xiao Xiao 1 , Jian Sun 1 , Zhitao Wang 1 , Bin Li 6
Affiliation  

The rAAV2-ND4 gene therapy that targets mt11778G>A was delivered to nine patients with Leber’s hereditary optic neuropathy (LHON) and continues to be safe and potentially effective at long-term (75–90 months) follow-up.



中文翻译:

Leber遗传性视神经病变的基因治疗七年随访。

靶向mt11778G> A的rAAV2-ND4基因治疗已被送至9名Leber遗传性视神经病变(LHON)患者,并且在长期(75-90个月)随访中仍是安全且可能有效的。

更新日期:2020-02-25
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