Matches in SemOpenAlex for { <https://semopenalex.org/work/W2054406966> ?p ?o ?g. }
- W2054406966 endingPage "619" @default.
- W2054406966 startingPage "613" @default.
- W2054406966 abstract "ABSTRACT Increased expression of fetal hemoglobin can ameliorate the clinical severity of sickle cell disease. Whereas temporary induction of fetal hemoglobin can be achieved by pharmacologic therapy, gene transfer resulting in high-level expression of the fetal γ-globin gene may provide a permanent cure for sickle cell disease. We had previously developed a high-titer, genetically stable retroviral vector in which the human γ-globin gene was linked to HS-40, the major regulatory element of the human α-globin gene cluster. Based on experience in transgenic mice, the truncated promoter of the γ-globin gene of this vector should be active in adult erythroid cells. Our earlier studies demonstrated that this retroviral vector can give rise to high-level expression of the human γ-globin gene in murine erythroleukemia (MEL) cells. We have now utilized this vector to transduce murine bone marrow cells that were transplanted into W/Wv recipient mice. Analysis of transduction of murine BFU-e's in vitro and peripheral blood cells from transplanted mice in vivo demonstrated efficient transfer of the human γ-globin gene. However, in contrast to the high level of expression of the human γ-globin gene of this vector in MEL cells, the gene was completely silent in vivo in all transplanted mice. These observations confirm that all the necessary regulatory elements responsible for the developmental stage-specific expression of the human γ-globin gene reside in its proximal sequences. They also emphasize the differences between gene regulation in MEL cells, transgenic mice, and retroviral gene transfer vectors. For this form of globin gene therapy to succeed, the proximal regulatory elements of the human γ-globin gene may have to be replaced with different regulatory elements that allow the expression of the γ-globin coding sequences in adult red cells in vivo." @default.
- W2054406966 created "2016-06-24" @default.
- W2054406966 creator A5010106063 @default.
- W2054406966 creator A5050368246 @default.
- W2054406966 creator A5086795238 @default.
- W2054406966 creator A5086865233 @default.
- W2054406966 date "2000-12-01" @default.
- W2054406966 modified "2023-09-27" @default.
- W2054406966 title "In Vivo Silencing of the Human γ-Globin Gene in Murine Erythroid Cells Following Retroviral Transduction" @default.
- W2054406966 cites W133981267 @default.
- W2054406966 cites W1515900859 @default.
- W2054406966 cites W176130659 @default.
- W2054406966 cites W1856681085 @default.
- W2054406966 cites W1872079788 @default.
- W2054406966 cites W188488539 @default.
- W2054406966 cites W1972406498 @default.
- W2054406966 cites W1977708103 @default.
- W2054406966 cites W1997086574 @default.
- W2054406966 cites W1997680183 @default.
- W2054406966 cites W2008533018 @default.
- W2054406966 cites W2008634652 @default.
- W2054406966 cites W2018552733 @default.
- W2054406966 cites W2033364271 @default.
- W2054406966 cites W2048404769 @default.
- W2054406966 cites W2067835109 @default.
- W2054406966 cites W2089305246 @default.
- W2054406966 cites W2102389958 @default.
- W2054406966 cites W2102707713 @default.
- W2054406966 cites W2104649610 @default.
- W2054406966 cites W2128006259 @default.
- W2054406966 cites W2151919704 @default.
- W2054406966 cites W2165300787 @default.
- W2054406966 cites W2182633717 @default.
- W2054406966 cites W2297467794 @default.
- W2054406966 cites W2305289606 @default.
- W2054406966 cites W2313541759 @default.
- W2054406966 cites W2324143840 @default.
- W2054406966 cites W2406030793 @default.
- W2054406966 cites W2409351985 @default.
- W2054406966 cites W2471265469 @default.
- W2054406966 cites W267085019 @default.
- W2054406966 cites W75372324 @default.
- W2054406966 doi "https://doi.org/10.1006/bcmd.2000.0343" @default.
- W2054406966 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/11358353" @default.
- W2054406966 hasPublicationYear "2000" @default.
- W2054406966 type Work @default.
- W2054406966 sameAs 2054406966 @default.
- W2054406966 citedByCount "26" @default.
- W2054406966 countsByYear W20544069662012 @default.
- W2054406966 countsByYear W20544069662013 @default.
- W2054406966 countsByYear W20544069662014 @default.
- W2054406966 countsByYear W20544069662015 @default.
- W2054406966 countsByYear W20544069662016 @default.
- W2054406966 crossrefType "journal-article" @default.
- W2054406966 hasAuthorship W2054406966A5010106063 @default.
- W2054406966 hasAuthorship W2054406966A5050368246 @default.
- W2054406966 hasAuthorship W2054406966A5086795238 @default.
- W2054406966 hasAuthorship W2054406966A5086865233 @default.
- W2054406966 hasConcept C102230213 @default.
- W2054406966 hasConcept C104317684 @default.
- W2054406966 hasConcept C111599444 @default.
- W2054406966 hasConcept C119056186 @default.
- W2054406966 hasConcept C125996951 @default.
- W2054406966 hasConcept C150194340 @default.
- W2054406966 hasConcept C15152581 @default.
- W2054406966 hasConcept C153911025 @default.
- W2054406966 hasConcept C172680121 @default.
- W2054406966 hasConcept C2776317666 @default.
- W2054406966 hasConcept C2779234561 @default.
- W2054406966 hasConcept C32470452 @default.
- W2054406966 hasConcept C40767141 @default.
- W2054406966 hasConcept C54355233 @default.
- W2054406966 hasConcept C55493867 @default.
- W2054406966 hasConcept C86803240 @default.
- W2054406966 hasConceptScore W2054406966C102230213 @default.
- W2054406966 hasConceptScore W2054406966C104317684 @default.
- W2054406966 hasConceptScore W2054406966C111599444 @default.
- W2054406966 hasConceptScore W2054406966C119056186 @default.
- W2054406966 hasConceptScore W2054406966C125996951 @default.
- W2054406966 hasConceptScore W2054406966C150194340 @default.
- W2054406966 hasConceptScore W2054406966C15152581 @default.
- W2054406966 hasConceptScore W2054406966C153911025 @default.
- W2054406966 hasConceptScore W2054406966C172680121 @default.
- W2054406966 hasConceptScore W2054406966C2776317666 @default.
- W2054406966 hasConceptScore W2054406966C2779234561 @default.
- W2054406966 hasConceptScore W2054406966C32470452 @default.
- W2054406966 hasConceptScore W2054406966C40767141 @default.
- W2054406966 hasConceptScore W2054406966C54355233 @default.
- W2054406966 hasConceptScore W2054406966C55493867 @default.
- W2054406966 hasConceptScore W2054406966C86803240 @default.
- W2054406966 hasIssue "6" @default.
- W2054406966 hasLocation W20544069661 @default.
- W2054406966 hasLocation W20544069662 @default.
- W2054406966 hasOpenAccess W2054406966 @default.
- W2054406966 hasPrimaryLocation W20544069661 @default.
- W2054406966 hasRelatedWork W2001489295 @default.
- W2054406966 hasRelatedWork W2006710959 @default.
- W2054406966 hasRelatedWork W204748332 @default.