RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice

Repair of β-globin pre-mRNA rendered defective by a thalassemia-causing splicing mutation, IVS2-654, in intron 2 of the human β-globin gene was accomplished in vivo in a mouse model of IVS2-654 thalassemia. This was effected by a systemically delivered splice-switching oligonucleotide (SSO), a morph...

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Veröffentlicht in:	Proceedings of the National Academy of Sciences - PNAS 2009-01, Vol.106 (4), p.1205-1210
Hauptverfasser:	Svasti, Saovaros, Suwanmanee, Thipparat, Fucharoen, Suthat, Moulton, Hong M, Nelson, Michelle H, Maeda, Nobuyo, Smithies, Oliver, Kole, Ryszard
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals beta-Globins - genetics Biological Sciences Blood Cell Shape - drug effects Cells DNA repair Erythrocytes - drug effects Erythrocytes - pathology Erythroid cells Hemoglobin Hemoglobins Hemoglobins - genetics Hemoglobins - metabolism Humans Injections, Intravenous Interleukin-12 - blood Messenger RNA Mice Mutation Mutation - genetics Oligomers Oligonucleotides Oligonucleotides - administration & dosage Oligonucleotides - adverse effects Oligonucleotides - pharmacology Peptides Reverse transcriptase polymerase chain reaction Ribonucleic acid RNA RNA Precursors - genetics RNA Precursors - metabolism RNA Splicing - drug effects RNA Splicing - genetics Rodents Splicing Thalassemia - blood Thalassemia - genetics Thalassemia - therapy
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