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A peptidomimetic siRNA transfection reagent forhighly effectivegene silencing

Journal Article · · Molecular BioSystems
DOI:https://doi.org/10.1039/b603229j· OSTI ID:918632
RNA interference (RNAi) techniques hold forth great promisefor therapeutic silencing of deleterious genes. However, clinicalapplications of RNAi require the development of safe and efficientmethods for intracellular delivery of small interfering RNA (siRNA)oligonucleotides specific to targeted genes. We describe the use of alipitoid, a cationic oligopeptoid phospholipid conjugate, for non-viraltransfection of synthetic siRNA oligos in cell culture. Thispeptidomimetic delivery vehicle allows for efficient siRNA transfectionin a variety of human cell lines with negligible toxicity and promotesextensive downregulation of the targeted genes at both the protein andthe mRNA level. We compare the lipitoid reagent to a standard commercialtransfection reagent. The lipitoid is highly efficient even in primaryIMR-90 human lung fibroblasts in which other commercial reagents aretypically ineffective.
Research Organization:
COLLABORATION - New YorkU.
DOE Contract Number:
AC02-05CH11231
OSTI ID:
918632
Report Number(s):
LBNL--61663
Journal Information:
Molecular BioSystems, Journal Name: Molecular BioSystems Vol. 2
Country of Publication:
United States
Language:
English

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