Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients.

Lacerra G; Sierakowska H; Carestia CFucharoen SSummerton JWeller DKole R

首页> 外文期刊>Proceedings of the National Academy of Sciences of the United States of America >Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients.

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Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients.

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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.

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《Proceedings of the National Academy of Sciences of the United States of America》 |2000年第17期|9591-9596|共6页
作者
Lacerra G; Sierakowska H; Carestia CFucharoen SSummerton JWeller DKole R;
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作者单位

Lineberger Comprehensive Cancer Center and Department of Pharmacology, University of North Carolina, Chapel Hill, NC 27599-7295, USA.;

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收录信息美国《科学引文索引》(SCI);美国《生物学医学文摘》(MEDLINE);美国《化学文摘》(CA);
原文格式 PDF
正文语种英语
中图分类自然科学总论;
关键词
beta-Thalassemia; Erythrocytes; Gene Therapy; Hemoglobin A; Oligonucleotides; Antisense; β地中海贫血; 红细胞; 基因疗法; 血红蛋白A;

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Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients.

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