http://www.cnr.it/ontology/cnr/individuo/prodotto/ID26945
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
- Pagina fine
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- 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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