Restoration of Human β-Globin Gene Expression in Murine and Human IVS2–654 Thalassemic Erythroid Cells by Free Uptake of Antisense Oligonucleotides-Reference-Cited by-同舟云学术

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Restoration of Human β-Globin Gene Expression in Murine and Human IVS2–654 Thalassemic Erythroid Cells by Free Uptake of Antisense Oligonucleotides

Published:2002-09-01 Issue:3 Volume:62 Page:545-553
ISSN:0026-895X
Container-title:Molecular Pharmacology
language:en
Short-container-title:Mol Pharmacol

Author:

Suwanmanee Thipparat,Sierakowska Halina,Lacerra Giuseppina,Svasti Saovaros,Kirby Suzanne,Walsh Christopher E.,Fucharoen Suthat,Kole Ryszard

Publisher

American Society for Pharmacology & Experimental Therapeutics (ASPET)

Subject

Pharmacology,Molecular Medicine

Reference35 articles.

1. β+ Thalassemia: Aberrant splicing results from a single point mutation in an intron

2. beta-Thalassemia in Chinese: use of in vivo RNA analysis and oligonucleotide hybridization in systematic characterization of molecular defects.

3. Reversibility of IVS 2 missplicing in a mutant human β-globin gene.;Dobkin;J Biol Chem,1985

4. Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides.

5. Phosphoramidate oligonucleotides as potent antisense molecules in cells and in vivo

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