目的:建立并验证rAAV2-ND4中可复制型AAV2(rcAAV2)的qPCR检测方法.方法:rAAV2-ND4样品(3.0×10^(10)vg·mL^(-1),每孔接种100μL)和wtAd5辅毒(6.0×10^(9)TCID50·mL^(-1),每孔接种10μL)同时感染HEK293细胞(6.0×105cells·mL^(-1),每孔1 mL接种至12孔板中,37℃和5%二氧化碳条件培养24 h),...
rAAV2 / 2- ND4(GS010)是一种重组复制缺陷型腺相关病毒,血清型2,其包含编码人野生型线粒体ND4蛋白的修饰互补DNA和用于将该蛋白易位到线粒体基质中的特异性MTS 。在该疾病的大鼠模型中,rAAV2 / 2- ND4已显示可有效挽救线粒体呼吸链复合体I的诱发缺陷(13),并且能够在培养的LHON中恢复复合体I活性和5'-三...
目的:建立并验证rAAV2-ND4中可复制型AAV2(rcAAV2)的qPCR检测方法.方法:rAAV2-ND4样品(3.0×10^(10)vg·mL^(-1),每孔接种100μL)和wtAd5辅毒(6.0×10^(9)TCID50·mL^(-1),每孔接种10μL)同时感染HEK293细胞(6.0×105cells·mL^(-1),每孔1 mL接种至12孔板中,37℃和5%二氧化碳条件培养24 h),...
Improvement in the contralateral eye may be explained by the transfer of rAAV2/2-ND4 to the sham-treated eye.Mark MosterAlfredo SadunThomas KlopstockNancy NewmanCatherine Vignal-ClermontValerio CarelliPatrick Yu-Wai-ManValerie BiousseRobert Sergott...
Amr K. HassanMaram MohsenHashem Abu Serhan
方法采用多中心前瞻性非随机对照临床试验研究设计,于2017年12月至2018年2月在十堰市太和医院眼科中心招募线粒体DNA 11778位点突变的LHON患者40例80眼,以视力较差眼或右眼(双眼视力相等时)行玻璃体腔内注射重组腺相关病毒2-还原型辅酶Ⅰ脱氢酶4(rAAV2-ND4),对侧眼作为未注射眼,并依此分为注射眼组和未注射眼组,...
patients with mutation at site 11778 are unknown.To analyse correlations between visual acuity prognosis and baseline characteristics of LHON after rAAV2-ND4 gene therapy.Retrospective study.Fifty-three LHON patients with a mutation at site 11778.Single-eye intravitreal injection of rAAV2-ND4.Sex, ...
After receiving an intravitreal injection of rAAV2-ND4, patients followed up over a certain time period showed a definite increase in visual acuity. Visual acuity testing is critical for assessing the efficacy of rAAV2-ND4 intravitreal injection....
83. Relationship Between Immune Responses and Ocular Inflammation: What Is Learnt from Intravitreal Injection of rAAV2-2-ND4 (GS010) in Non-Human Primates and Leber Hereditary Optic Neuropathy (LHON) Patientsdoi:10.1016/s1525-0016(16)32892-1Céline Bouquet...
Methods: Single‐eye intravitreal injection of rAAV2‐ND4. Main Outcome Measures: Sex, onset age, duration of disease, best‐corrected visual acuity (BCVA), visual field index (VFI) and mean deviation (MD) were recorded for all patients at baseline. BCVA was recorded at 1‐ and 3‐month ...