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: Saovaros Svasti, Thipparat Suwanmanee, Suthat Fucharoen, Hong M. Moulton, Michelle H. Nelson, Nobuyo Maeda, Oliver Smithies, Ryszard Kole
Other Authors: The University of North Carolina at Chapel Hill
Format: Article
Published: 2018
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Online Access:https://repository.li.mahidol.ac.th/handle/123456789/28397
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spelling th-mahidol.283972018-09-13T14:17:45Z RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice Saovaros Svasti Thipparat Suwanmanee Suthat Fucharoen Hong M. Moulton Michelle H. Nelson Nobuyo Maeda Oliver Smithies Ryszard Kole The University of North Carolina at Chapel Hill The Institute of Science and Technology for Research and Development, Mahidol University AVI BioPharma Incorporated Multidisciplinary 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 morpholino oligomer conjugated to an arginine-rich peptide. The SSO blocked the aberrant splice site in the targeted pre-mRNA and forced the splicing machinery to reselect existing correct splice sites. Repaired β-globin mRNA restored significant amounts of hemoglobin in the peripheral blood of the IVS2-654 mouse, improving the number and quality of erythroid cells. © 2009 by The National Academy of Sciences of the USA. 2018-09-13T07:17:45Z 2018-09-13T07:17:45Z 2009-01-27 Article Proceedings of the National Academy of Sciences of the United States of America. Vol.106, No.4 (2009), 1205-1210 10.1073/pnas.0812436106 10916490 00278424 2-s2.0-59049089272 https://repository.li.mahidol.ac.th/handle/123456789/28397 Mahidol University SCOPUS https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=59049089272&origin=inward
institution Mahidol University
building Mahidol University Library
continent Asia
country Thailand
Thailand
content_provider Mahidol University Library
collection Mahidol University Institutional Repository
topic Multidisciplinary
spellingShingle Multidisciplinary
Saovaros Svasti
Thipparat Suwanmanee
Suthat Fucharoen
Hong M. Moulton
Michelle H. Nelson
Nobuyo Maeda
Oliver Smithies
Ryszard Kole
RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice
description 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 morpholino oligomer conjugated to an arginine-rich peptide. The SSO blocked the aberrant splice site in the targeted pre-mRNA and forced the splicing machinery to reselect existing correct splice sites. Repaired β-globin mRNA restored significant amounts of hemoglobin in the peripheral blood of the IVS2-654 mouse, improving the number and quality of erythroid cells. © 2009 by The National Academy of Sciences of the USA.
author2 The University of North Carolina at Chapel Hill
author_facet The University of North Carolina at Chapel Hill
Saovaros Svasti
Thipparat Suwanmanee
Suthat Fucharoen
Hong M. Moulton
Michelle H. Nelson
Nobuyo Maeda
Oliver Smithies
Ryszard Kole
format Article
author Saovaros Svasti
Thipparat Suwanmanee
Suthat Fucharoen
Hong M. Moulton
Michelle H. Nelson
Nobuyo Maeda
Oliver Smithies
Ryszard Kole
author_sort Saovaros Svasti
title RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice
title_short RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice
title_full RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice
title_fullStr RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice
title_full_unstemmed RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice
title_sort rna repair restores hemoglobin expression in ivs2-654 thalassemic mice
publishDate 2018
url https://repository.li.mahidol.ac.th/handle/123456789/28397
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