Retrovirus-mediated transfer of the human glucocerebrosidase gene to Gaucher fibroblasts.
Retrovirus-mediated transfer of the human glucocerebrosidase gene to Gaucher fibroblasts.
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逆转录病毒介导的人葡萄糖脑苷脂酶基因向戈谢成纤维细胞的转移。
DOI：
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发表时间：
1986
期刊：
影响因子：
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通讯作者：
E. Ginns
中科院分区：
文献类型：
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作者：
P. Choudary;J. Barranger;S. Tsuji;J. Mayor;M. Lamarca;C. Cepko;R. Mulligan;E. Ginns
构建了一种含有人葡萄糖脑苷酶完整编码序列的互补DNA的重组逆转录病毒，并将其用于感染小鼠细胞和2型戈谢病患者的人成纤维细胞。cDNA稳定地整合在受体细胞的基因组中。感染的细胞产生葡萄糖脑苷酶交叉反应物质，蛋白质被糖基化，并含有2型突变型葡萄糖脑苷酶缺失的表位。本研究证明了以逆转录病毒为载体将编码葡萄糖脑苷酶的基因高效转移到戈谢氏细胞的可行性。
A recombinant retrovirus carrying complementary DNA containing the complete coding sequence for human glucocerebrosidase was constructed and used to infect mouse cells and human fibroblasts from a type 2 Gaucher patient. The cDNA stably integrated in the genome of the recipient cell. Infected cells made glucocerebrosidase-cross-reacting material, and the protein was glycosylated and contained the epitope(s) missing in the type 2 mutant glucocerebrosidase. This study demonstrates the feasibility of efficiently transferring the gene encoding glucocerebrosidase to Gaucher cells using retrovirus as vector.