Vision 1 year after gene therapy for Leber's congenital amaurosis.
作者信息
Cideciyan Artur V, Hauswirth William W, Aleman Tomas S, Kaushal Shalesh, Schwartz Sharon B, Boye Sanford L, Windsor Elizabeth A M, Conlon Thomas J, Sumaroka Alexander, Roman Alejandro J, Byrne Barry J, Jacobson Samuel G
出版信息
N Engl J Med. 2009 Aug 13;361(7):725-7. doi: 10.1056/NEJMc0903652.
Abstract
摘要
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本文引用的文献
1
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics.针对RPE65异构酶缺乏症的人类基因疗法可激活视觉的视黄醛循环，但视杆动力学缓慢。
Proc Natl Acad Sci U S A. 2008 Sep 30;105(39):15112-7. doi: 10.1073/pnas.0807027105. Epub 2008 Sep 22.
2
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.通过眼内视网膜下注射腺相关病毒基因载体治疗由RPE65基因突变引起的莱伯先天性黑蒙：I期试验的短期结果
Hum Gene Ther. 2008 Oct;19(10):979-90. doi: 10.1089/hum.2008.107.
3
Leber congenital amaurosis: genes, proteins and disease mechanisms.莱伯先天性黑蒙：基因、蛋白质与疾病机制
Prog Retin Eye Res. 2008 Jul;27(4):391-419. doi: 10.1016/j.preteyeres.2008.05.003. Epub 2008 Jun 1.
4
Effect of gene therapy on visual function in Leber's congenital amaurosis.基因治疗对莱伯先天性黑蒙视觉功能的影响。
N Engl J Med. 2008 May 22;358(21):2231-9. doi: 10.1056/NEJMoa0802268. Epub 2008 Apr 27.
5
Safety and efficacy of gene transfer for Leber's congenital amaurosis.基因转移治疗莱伯先天性黑蒙的安全性和有效性。
N Engl J Med. 2008 May 22;358(21):2240-8. doi: 10.1056/NEJMoa0802315. Epub 2008 Apr 27.
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