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首页> 外文期刊>Proceedings of the National Academy of Sciences of the United States of America >PHENOTYPE CORRECTION IN RETINAL PIGMENT EPITHELIUM IN MURINE MUCOPOLYSACCHARIDOSIS VII BY ADENOVIRUS-MEDIATED GENE TRANSFER
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PHENOTYPE CORRECTION IN RETINAL PIGMENT EPITHELIUM IN MURINE MUCOPOLYSACCHARIDOSIS VII BY ADENOVIRUS-MEDIATED GENE TRANSFER

机译：腺病毒介导的基因转移修复鼠黏膜多糖糖蛋白VII视网膜色素上皮中的表型

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摘要

We have studied the use of adenovirus-mediated gene transfer to reverse the pathologic changes of lysosomal storage disease caused by beta-glucuronidase deficiency in the eyes of mice with mucopolysaccharidosis VII. A recombinant adenovirus carrying the human beta-glucuronidase cDNA coding region under the control of a non-tissue-specific promoter was injected intravitreally or subretinally into the eyes of mice with mucopolysaccharidosis VII. At 1-3 weeks after injection, the treated and control eyes were examined histochemically for beta-glucuronidase expression and histologically for phenotypic correction of the lysosomal storage defect. Enzymatic expression was detected 1-3 weeks after injection. Storage vacuoles in the retinal pigment epithelium (RPE) were still present 1 week after gene transfer but were reduced to undetectable levels by 3 weeks in both intravitreally and subretinally injected eyes. There was minimal evidence of ocular pathology associated with the viral injection. These data indicate that adenovirus-mediated gene transfer to the eye may provide for adjunctive therapy for lysosomal storage diseases affecting the RPE in conjunction with enzyme replacement and/or gene therapies for correction of systemic disease manifestations. The data also support the view that recombinant adenovirus may be useful as a gene therapy vector for retinal degenerations that result from a primary genetic defect in the RPE cells. [References: 30]
机译：我们已经研究了使用腺病毒介导的基因转移来逆转由粘多糖贮积症VII引起的小鼠眼中由β-葡萄糖醛酸苷酶缺乏引起的溶酶体贮积病的病理变化。在非组织特异性启动子的控制下，将携带人β-葡糖醛酸糖苷酶cDNA编码区的重组腺病毒玻璃体内或视网膜下注射到患有粘多糖贮积症VII的小鼠的眼睛中。注射后1-3周，对治疗和对照眼进行组织化学检查，检查β-葡萄糖醛酸苷酶的表达，并在组织学上检查溶酶体贮藏缺陷的表型。注射后1-3周检测到酶促表达。基因转移后1周，视网膜色素上皮（RPE）中的储存液泡仍然存在，但在玻璃体内和视网膜下注射的眼中，这些液泡在3周前已降至无法检测的水平。几乎没有证据表明与病毒注射有关的眼部病理。这些数据表明，腺病毒介导的基因转移到眼睛可为影响RPE的溶酶体贮积病提供辅助治疗，并结合酶替代和/或基因疗法来纠正全身性疾病表现。数据还支持这样的观点，即重组腺病毒可以用作因RPE细胞中的原始遗传缺陷而导致的视网膜变性的基因治疗载体。 [参考：30]

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