期刊
NUCLEIC ACID THERAPEUTICS
卷 26, 期 6, 页码 381-391出版社
MARY ANN LIEBERT, INC
DOI: 10.1089/nat.2016.0631
关键词
reporter cell lines; splice-switching oligonucleotides; pLuc 705; delivery vectors; gymnotic delivery; oligonucleotide chemistries
资金
- Swedish Medical Research Council
- Swedish Research Council
- Swedish Society of Medical Research (SSMF)
New advances in oligonucleotide (ON) chemistry emerge continuously, and over the last few years, several aspects of ON delivery have been improved. However, clear knowledge regarding how certain chemistries behave alone, or in combination with various delivery vectors, is limited. Moreover, characterization is frequently limited to a single reporter cell line and, when different cell types are studied, experiments are commonly not carried out under similar conditions, hampering comparative analysis. To address this, we have developed a small tissue library of new, stable, pLuc/705 splice-switching reporter cell lines (named HuH7_705, U-2 OS_705, C2C12_705, and Neuro-2a_705). Our data show that, indeed, the cell type used in activity screenings influences the efficiency of ONs of different chemistry (phosphorothioate with locked nucleic acid or 2-O-methyl with or without N,N-diethyl-4-(4-nitronaphthalen-1-ylazo)-phenylamine). Likewise, the delivery method, Lipofectamine((R)) 2000, PepFect14 nanoparticles, or naked uptake, also demonstrates cell-type-dependent outcomes. Taken together, these cell lines can potentially become useful tools for future in vitro evaluation of new nucleic acid-based oligomers as well as delivery compounds for splice-switching approaches and cell-specific therapies.
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