http://www.cnr.it/ontology/cnr/individuo/prodotto/ID26488
Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides (Articolo in rivista)
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- Label
- Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides (Articolo in rivista) (literal)
- Anno
- 2002-01-01T00:00:00+01:00 (literal)
- Alternative label
Suwanmanee T, Sierakowska H, Lacerra G, Svasti S, Kirby S, Walsh CE, Fucharoen S, Kole R (2002)
Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides
in Molecular pharmacology (Print)
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- Suwanmanee T, Sierakowska H, Lacerra G, Svasti S, Kirby S, Walsh CE, Fucharoen S, Kole R (literal)
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- ISI Web of Science (WOS) (literal)
- Titolo
- Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides (literal)
- Abstract
- Correct human beta-globin mRNA has been restored in erythroid
cells from transgenic mice carrying the human gene with beta-globin
IVS2-654 splice mutation and from thalassemia patients with the
IVS2-654/beta(E) genotype. This was accomplished in a dose- and
time-dependent manner by free uptake of morpholino oligonucleotide
antisense to the aberrant splice site at position 652 of intron 2 in
beta-globin pre-mRNA. Under optimal conditions of oligonucleotide
uptake, the maximal levels of correct human beta-globin mRNA and
hemoglobin A in patients' erythroid cells were 77 and 54%,
respectively. These levels of correction were equal to, if not higher
than, those obtained by syringe loading of the oligonucleotide into the
cells.
Comparison of splicing correction results with the cellular uptake of
fluorescein-labeled oligonucleotide indicated that the levels of mRNA
and hemoglobin A correlate well with the nuclear localization of the
oligonucleotide and the degree of erythroid differentiation of cultured
cells. Similar but not as pronounced results were obtained after the
oligonucleotide treatment of bone marrow cells from IVS2-654
mouse. The effectiveness of the free antisense morpholino
oligonucleotide in restoration of correct splicing of IVS2-654
pre-mRNA in cultured erythropoietic cells from transgenic mice and
thalassemic patients suggests the applicability of this or similar
compounds in in vivo experiments and possibly in treatment of
thalassemia.
(literal)
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