Recombinant Adeno-associated Viral Vectors Serotypes 6 and 9 are Able to Transduce Human Tracheal Epithelial Cells but Not Human Induced Pluripotent Stem Cells

被引：3

作者：

Belova, L. ^{[1
]}

Demchenko, A. ^{[1
]}

Kochergin-Nikitsky, K. ^{[1
]}

Kondrateva, E. ^{[1
]}

Slesarenko, Ya. ^{[1
]}

Salikhova, D. ^{[1
]}

Lavrov, A. ^{[1
]}

Efremova, A. ^{[1
]}

Bukharova, T. ^{[1
]}

Goldshtein, D. ^{[1
]}

Smirnikhina, S. ^{[1
]}

机构：

[1] Res Ctr Med Genet, Moskvorechye 1, Moscow 115478, Russia

来源：

MOLECULAR BIOTECHNOLOGY | 2023年 / 65卷 / 09期

关键词：

Adeno-associated viral vectors; Cell line; CFTE29o-; hiPSCs; Cystic fibrosis; EFFICIENT TRANSDUCTION; CYSTIC-FIBROSIS; GENE-TRANSFER; LUNG; ORGANOIDS; REPAIR; AAV5;

D O I：

10.1007/s12033-023-00668-4

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Recombinant adeno-associated viruses (rAAVs) may be useful for the development of gene therapy for hereditary diseases. Patient-specific human induced pluripotent stem cells (hiPSCs) can be differentiated into a variety of cells which are difficult or impossible to obtain by biopsy. To date, few research on the efficiency of rAAV transduction of hiPSCs has been published, but the obtained data are very contradictory and do not answer the actual question: how effective are rAAVs for the delivery of transgenes into hiPSCs. In this work, we used rAAV serotypes 5, 6, and 9 carrying the GFP transgene. The transduction efficiency of rAAV2/9-GFP and rAAV2/6-GFP for the immortalized tracheal epithelial cell line derived from a patient with cystic fibrosis (CFTE29o-) was relatively high. At the same time, the efficiency of transduction of iPSCs from a healthy donor and a cystic fibrosis (CF) donor was extremely low. Thus, our results show that the efficiency of hiPSC transduction by rAAV serotypes 5, 6, and 9 is not suitable for the delivery of transgenes.

引用

页码：1539 / 1546

页数：8

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