FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2050347612> ?p ?o ?g. }

• W2050347612 endingPage "615" @default.
• W2050347612 startingPage "607" @default.
• W2050347612 abstract "We previously compared the expression of several human factor VIII (fVIII) transgene variants and demonstrated the superior expression properties of B domain-deleted porcine fVIII. Subsequently, a hybrid human/porcine fVIII molecule (HP-fVIII) comprising 91% human amino-acid sequence was engineered to maintain the high-expression characteristics of porcine fVIII. The bioengineered construct then was used effectively to treat knockout mice with hemophilia A. In the current study, we focused on optimizing self-inactivating (SIN) lentiviral vector systems by analyzing the efficacy of various lentiviral components in terms of virus production, transduction efficiency and transgene expression. Specifically, three parameters were evaluated: (1) the woodchuck hepatitis post-transcriptional regulatory element (WPRE), (2) HIV versus SIV viral vector systems and (3) various internal promoters. The inclusion of a WPRE sequence had negligible effects on viral production and HP-fVIII expression. HIV and SIV vectors were compared and found to be similar with respect to transduction efficiency in both K562s and HEK-293T cells. However, there was an enhanced expression of HP-fVIII by the SIV system, which was evident in both K562 and BHK-M cell lines. To further compare expression of HP-fVIII from an SIV-based lentiviral system, we constructed expression vectors containing the high expression transgene and a human elongation factor-1 alpha, cytomegalovirus (CMV) or phosphoglycerate kinase promoter. Expression was significantly greater from the CMV promoter, which also yielded therapeutic levels of HP-fVIII in hemophilia A mice. Based on these studies, an optimized vector contains the HP-fVIII transgene driven by a CMV internal promoter within a SIV-based lentiviral backbone lacking a WPRE." @default.
• W2050347612 created "2016-06-24" @default.
• W2050347612 creator A5040467558 @default.
• W2050347612 creator A5051530484 @default.
• W2050347612 creator A5065987693 @default.
• W2050347612 creator A5086198464 @default.
• W2050347612 date "2012-09-20" @default.
• W2050347612 modified "2023-09-26" @default.
• W2050347612 title "Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A" @default.
• W2050347612 cites W1637867377 @default.
• W2050347612 cites W1769569140 @default.
• W2050347612 cites W1835972708 @default.
• W2050347612 cites W1964975805 @default.
• W2050347612 cites W1967531174 @default.
• W2050347612 cites W1983041761 @default.
• W2050347612 cites W1985656844 @default.
• W2050347612 cites W1986993620 @default.
• W2050347612 cites W1995004137 @default.
• W2050347612 cites W1995076136 @default.
• W2050347612 cites W2002228979 @default.
• W2050347612 cites W2004480433 @default.
• W2050347612 cites W2009761723 @default.
• W2050347612 cites W2010216896 @default.
• W2050347612 cites W2017444975 @default.
• W2050347612 cites W2023159885 @default.
• W2050347612 cites W2028813295 @default.
• W2050347612 cites W2037303802 @default.
• W2050347612 cites W2041157989 @default.
• W2050347612 cites W2041235125 @default.
• W2050347612 cites W2042700672 @default.
• W2050347612 cites W2045995615 @default.
• W2050347612 cites W2048182307 @default.
• W2050347612 cites W2048767675 @default.
• W2050347612 cites W2052207235 @default.
• W2050347612 cites W2058518705 @default.
• W2050347612 cites W2061645642 @default.
• W2050347612 cites W2062321149 @default.
• W2050347612 cites W2063056703 @default.
• W2050347612 cites W2079827600 @default.
• W2050347612 cites W2089865394 @default.
• W2050347612 cites W2096643836 @default.
• W2050347612 cites W2097933470 @default.
• W2050347612 cites W2104531050 @default.
• W2050347612 cites W2111967568 @default.
• W2050347612 cites W2112771233 @default.
• W2050347612 cites W2128435780 @default.
• W2050347612 cites W2131731195 @default.
• W2050347612 cites W2133907434 @default.
• W2050347612 cites W2136274459 @default.
• W2050347612 cites W2137440275 @default.
• W2050347612 cites W2141570423 @default.
• W2050347612 cites W2142439096 @default.
• W2050347612 cites W2148920506 @default.
• W2050347612 cites W2153867928 @default.
• W2050347612 cites W2400075822 @default.
• W2050347612 doi "https://doi.org/10.1038/gt.2012.76" @default.
• W2050347612 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/3552131" @default.
• W2050347612 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/22996197" @default.
• W2050347612 hasPublicationYear "2012" @default.
• W2050347612 type Work @default.
• W2050347612 sameAs 2050347612 @default.
• W2050347612 citedByCount "31" @default.
• W2050347612 countsByYear W20503476122013 @default.
• W2050347612 countsByYear W20503476122014 @default.
• W2050347612 countsByYear W20503476122015 @default.
• W2050347612 countsByYear W20503476122016 @default.
• W2050347612 countsByYear W20503476122017 @default.
• W2050347612 countsByYear W20503476122018 @default.
• W2050347612 countsByYear W20503476122019 @default.
• W2050347612 countsByYear W20503476122020 @default.
• W2050347612 countsByYear W20503476122021 @default.
• W2050347612 countsByYear W20503476122022 @default.
• W2050347612 crossrefType "journal-article" @default.
• W2050347612 hasAuthorship W2050347612A5040467558 @default.
• W2050347612 hasAuthorship W2050347612A5051530484 @default.
• W2050347612 hasAuthorship W2050347612A5065987693 @default.
• W2050347612 hasAuthorship W2050347612A5086198464 @default.
• W2050347612 hasBestOaLocation W20503476121 @default.
• W2050347612 hasConcept C100012643 @default.
• W2050347612 hasConcept C102230213 @default.
• W2050347612 hasConcept C104317684 @default.
• W2050347612 hasConcept C111599444 @default.
• W2050347612 hasConcept C15152581 @default.
• W2050347612 hasConcept C153911025 @default.
• W2050347612 hasConcept C159047783 @default.
• W2050347612 hasConcept C174510640 @default.
• W2050347612 hasConcept C2522874641 @default.
• W2050347612 hasConcept C2780593183 @default.
• W2050347612 hasConcept C2780617729 @default.
• W2050347612 hasConcept C2781079990 @default.
• W2050347612 hasConcept C32470452 @default.
• W2050347612 hasConcept C33161422 @default.
• W2050347612 hasConcept C40767141 @default.
• W2050347612 hasConcept C54355233 @default.
• W2050347612 hasConcept C55493867 @default.
• W2050347612 hasConcept C81885089 @default.
• W2050347612 hasConcept C86803240 @default.
• W2050347612 hasConcept C92087593 @default.

• next