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基于猪腺相关病毒血清型的重组载体可转导鼠和猪的视网膜。

Recombinant vectors based on porcine adeno-associated viral serotypes transduce the murine and pig retina.

机构信息

Telethon Institute of Genetics and Medicine, Napoli, Italy.

出版信息

PLoS One. 2013;8(3):e59025. doi: 10.1371/journal.pone.0059025. Epub 2013 Mar 8.

DOI:10.1371/journal.pone.0059025
PMID:23520549
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3592811/
Abstract

Recombinant adeno-associated viral (AAV) vectors are known to safely and efficiently transduce the retina. Among the various AAV serotypes available, AAV2/5 and 2/8 are the most effective for gene transfer to photoreceptors (PR), which are the most relevant targets for gene therapy of inherited retinal degenerations. However, the search for novel AAV serotypes with improved PR transduction is ongoing. In this work we tested vectors derived from five AAV serotypes isolated from porcine tissues (referred to as porcine AAVs, four of which are newly identified) for their ability to transduce both the murine and the cone-enriched pig retina. Porcine AAV vectors expressing EGFP under the control of the CMV promoter were injected subretinally either in C57BL/6 mice or Large White pigs. The resulting retinal tropism was analyzed one month later on histological sections, while levels of PR transduction were assessed by Western blot. Our results show that all porcine AAV transduce murine and porcine retinal pigment epithelium and PR upon subretinal administration. AAV2/po1 and 2/po5 are the most efficient porcine AAVs for murine PR transduction and exhibit the strongest tropism for pig cone PR. The levels of PR transduction obtained with AAV2/po1 and 2/po5 are similar, albeit not superior, to those obtained with AAV2/5 and AAV2/8, which evinces AAV2/po1 and 2/po5 to be promising vectors for retinal gene therapy.

摘要

重组腺相关病毒（AAV）载体已知可安全有效地转导视网膜。在可用的各种 AAV 血清型中，AAV2/5 和 2/8 是向光感受器（PR）进行基因转移最有效的，PR 是遗传性视网膜变性基因治疗的最相关靶标。然而，仍在寻找具有改善 PR 转导能力的新型 AAV 血清型。在这项工作中，我们测试了源自猪组织的五种 AAV 血清型（称为猪 AAV，其中四种是新鉴定的）的载体，以评估它们向小鼠和富含锥体的猪视网膜转导的能力。受 CMV 启动子控制表达 EGFP 的猪 AAV 载体通过视网膜下注射到 C57BL/6 小鼠或大白猪中。一个月后，通过组织学切片分析视网膜的靶向性，同时通过 Western blot 评估 PR 转导水平。我们的结果表明，所有猪 AAV 在视网膜下给药后均可转导小鼠和猪视网膜色素上皮和 PR。AAV2/po1 和 2/po5 是用于小鼠 PR 转导的最有效的猪 AAV，对猪锥体 PR 具有最强的靶向性。用 AAV2/po1 和 2/po5 获得的 PR 转导水平与用 AAV2/5 和 AAV2/8 获得的水平相似，尽管不优越，这表明 AAV2/po1 和 2/po5 是有前途的视网膜基因治疗载体。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6727/3592811/1023694ff2fd/pone.0059025.g008.jpg
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