Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients. (Articolo in rivista)

Type
Label
  • Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients. (Articolo in rivista) (literal)
Anno
  • 2000-01-01T00:00:00+01:00 (literal)
Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#doi
  • 10.1073/pnas.97.17.9591 (literal)
Alternative label
  • Lacerra G, Sierakowska H, Carestia C, Fucharoen S, Summerton J, Weller D, Kole R. (2000)
    Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients.
    in Proceedings of the National Academy of Sciences of the United States of America
    (literal)
Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#autori
  • Lacerra G, Sierakowska H, Carestia C, Fucharoen S, Summerton J, Weller D, Kole R. (literal)
Pagina inizio
  • 9591 (literal)
Pagina fine
  • 9596 (literal)
Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#numeroVolume
  • 97 (literal)
Rivista
Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#pagineTotali
  • 6 (literal)
Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#numeroFascicolo
  • 17 (literal)
Note
  • ISI Web of Science (WOS) (literal)
Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#affiliazioni
  • *Lineberger Comprehensive Cancer Center and Department of Pharmacology, University of North Carolina, Chapel Hill, NC 27599-7295; +Istituto Internazionale di Genetica e Biofisica-Consiglia Nazionale delle Ricerche, Naples, Italy; ?Institute of Biochemistry and Biophysics, Polish Academy of Sciences, 02-106 Warsaw, Poland; §Thalassemia Research Center, Mahidol University, Nakornpathom, Thailand; ¶GeneTools, Corvallis, OR 97339; and iAVI Biopharma, Portland, OR 97258 (literal)
Titolo
  • Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients. (literal)
Abstract
  • Mononuclear cells from peripheral blood of thalassemic patients were treated with morpholino oligonucleotides antisense to aberrant splice sites in mutant beta-globin precursor mRNAs (pre-mRNAs). The oligonucleotides restored correct splicing and translation of beta-globin mRNA, increasing the hemoglobin (Hb) A synthesis in erythroid cells from patients with IVS2-654/beta(E), IVS2-745/IVS2-745, and IVS2-745/IVS2-1 genotypes. The maximal Hb A level for repaired IVS2-745 mutation was approximately 30% of normal; Hb A was still detectable 9 days after a single treatment with oligonucleotide. Thus, expression of defective beta-globin genes was repaired and significant level of Hb A was restored in a cell population that would be targeted in clinical applications of this approach. (literal)
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