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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Main Authors: Svasti, Saovaros, Suwanmanee, Thipparat, Fucharoen, Suthat, Moulton, Hong M., Nelson, Michelle H., Maeda, Nobuyo, Smithies, Oliver, Kole, Ryszard
Formato: Artigo
Idioma:Inglês
Publicado: National Academy of Sciences 2009
Assuntos:
Biological Sciences
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC2633555/
https://ncbi.nlm.nih.gov/pubmed/19164558
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0812436106
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