Preferred Label : Bifunctional Expression Vector Plasmid DNA-bi-shRNA EWS/FLI1 Type 1 Lipoplex;
NCIt synonyms : Pbi-functional Short Hairpin RNA EWS/FLI1 Type 1 Lipoplex; Pbi-functional shRNA EWS-FLI1 Type 1 Lipoplex; pbi-shRNA EWS/FLI1 Type 1 LP; pbi-shRNA EWS/FLI1 Type 1 LPX; Pbi-shRNA EWS/FLI1 Type 1 Lipoplex;
NCIt definition : A proprietary plasmid DNA expression vector encoding bi-functional short hairpin RNAs
(bi-shRNAs) targeting the identical type 1 translocation junction region of the human
fusion oncogene Ewing sarcoma (EWS)/Ets family transcription factor Friend leukemia
virus integration 1 (FLI1) and are encapsulated in liposomal delivery vehicle (lipoplex;
LPX), with potential antineoplastic activity. pbi-shRNA EWS/FLI1 type 1 contains 2
stem-loop structures encoded by a plasmid vector: one cleavage-dependent unit with
perfectly matched passenger- and guide-strand, which is the small interfering RNA
(siRNA)-like component, and one cleavage-independent unit composed of a strategically
mismatched double strand, which is the microRNA (miRNA)-like component. Upon intratumoral
administration and transcription into tumor cells, one shRNA unit with an imperfectly
matched sequence causes inhibition of EWS/FLI1 messenger RNA (mRNA) translation (through
mRNA sequestration and cleavage-independent degradation) while the other unit with
a perfectly matched sequence promotes EWS/FLI1 mRNA degradation (through cleavage-dependent
mRNA silencing). This prevents EWS/FLI1 expression in tumor cells, which results in
a reduction of tumor cell proliferation. The EWS/FLI1 type 1 fusion gene product is
overexpressed in type 1 Ewing's sarcoma and correlates with increased tumor proliferation
and poor prognosis.;
NCI Metathesaurus CUI : CL507911;
Origin ID : C127121;
UMLS CUI : C4289921;
Semantic type(s)
- concept_is_in_subset
- has_target
