Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients
AUTOR(ES)
Lacerra, Giuseppina
FONTE
The National Academy of Sciences
RESUMO
Mononuclear cells from peripheral blood of thalassemic patients were treated with morpholino oligonucleotides antisense to aberrant splice sites in mutant β-globin precursor mRNAs (pre-mRNAs). The oligonucleotides restored correct splicing and translation of β-globin mRNA, increasing the hemoglobin (Hb) A synthesis in erythroid cells from patients with IVS2–654/βE, IVS2–745/IVS2–745, and IVS2–745/IVS2–1 genotypes. The maximal Hb A level for repaired IVS2–745 mutation was ≈30% of normal; Hb A was still detectable 9 days after a single treatment with oligonucleotide. Thus, expression of defective β-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.
ACESSO AO ARTIGO
http://www.pubmedcentral.nih.gov/articlerender.fcgi?artid=16909Documentos Relacionados
- Hemoglobin synthesis in cultures of hepatic erythroid cells from the human fetus
- Translational Control of Hemoglobin Synthesis in Thalassemic Bone Marrow
- Hemoglobin switching in sheep and goats: induction of hemoglobin C synthesis in cultures of sheep fetal erythroid cells.
- DIFFERENTIATION OF MAMMALIAN SOMATIC CELLS: DNA AND HEMOGLOBIN SYNTHESIS IN FETAL MOUSE YOLK SAC ERYTHROID CELLS*
- Regulation of Hemoglobin β-Chain Synthesis in Bone Marrow Erythroid Cells by α Chains