Homo sapiens
Ontology highlight
ABSTRACT:
PROVIDER: PRJNA277806 | ENA |
REPOSITORIES: ENA
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Project description:While small interfering RNAs (siRNAs) have been rapidly appreciated to induce gene silencing, efficient vectors for primary cells and for systemic in vivo delivery are lacking. We here present a novel chemically modified cell-penetrating peptide named PepFect6 (PF6) for efficient delivery of siRNAs into various types of cells including e.g. lymphocyte suspension cells and primary embryonic stem cells. Stable PF6/siRNA nano- particles rapidly enter entire cell populations resulting in strong and persistent RNAi responses, without associated transcriptomic or proteomic changes. In contrast to the majority of chemical reagents, PF6-mediated delivery is independent of cell confluence and, in most cases, not significantly hampered by the presence of serum. Finally, strong RNAi responses are observed in two different in vivo models following systemic delivery of PF6/siRNA in mice. Taken together, PF6 is an efficient siRNA delivery vector with superior delivery properties as compared to other tested transfection reagents.
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Project description:RNA interference (RNAi) holds tremendous potential as a therapeutic approach, especially in the treatment of malignant tumors. However, efficient and biocompatible delivery methods are needed for systemic delivery of siRNA. To achieve this goal, we have established a novel formulation of siRNA by incorporating it into reconstituted high density lipoprotein (rHDL) nanoparticles. Here, we demonstrate that rHDL nanoparticles facilitate highly efficient systemic delivery of siRNA in vivo, mediated by the scavenger receptor type B1 (SR-B1). Moreover, in therapeutic proof-of-concept studies, these nanoparticles were effective in targeting either signal transducer and activator of transcription 3 (STAT3) or focal adhesion kinase (FAK) expression in orthotopic mouse models of ovarian and colorectal cancer. These data indicate that an rHDL nanoparticle is a novel and highly efficient siRNA carrier, and therefore this novel technology could serve as the foundation for new cancer therapeutic approaches. To identify the role of STAT3 in ovarian cancer cell, we performed microarray after knocking down STAT3 in ovarican cancer cells (3 siCon and 3 siSTAT3).
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Project description:Despite the overwhelming information about sRNAs, one of the biggest challenges in the sRNA field is characterizing sRNA targetomes. Thus, we develop a novel method to identify RNAs that interact with a specific sRNA, regardless of the type of regulation (positive or negative) or targets (mRNA, tRNA, sRNA). This method is called MAPS: MS2 affinity purification coupled with RNA sequencing. As proof of principle, we identified RNAs bound to RyhB, a well-characterized E. coli sRNA. Identification of RNAs co-purified with MS2-RyhB in a rne131 ?ryhB strain. RyhB (without MS2) was used as control
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