Publication: Engineered U7 Small Nuclear RNA Restores Correct β-Globin Pre-mRNA Splicing in Mouse β<sup>iVS2-654</sup>-Thalassemic Erythroid Progenitor Cells
| dc.contributor.author | Annette D'Arqom | en_US |
| dc.contributor.author | Tiwaporn Nualkaew | en_US |
| dc.contributor.author | Natee Jearawiriyapaisarn | en_US |
| dc.contributor.author | Ryszard Kole | en_US |
| dc.contributor.author | Saovaros Svasti | en_US |
| dc.contributor.other | Universitas Airlangga | en_US |
| dc.contributor.other | The University of North Carolina at Chapel Hill | en_US |
| dc.contributor.other | Mahidol University | en_US |
| dc.contributor.other | Institute of Molecular Biosciences, Mahidol University | en_US |
| dc.contributor.other | Graduate Program in Molecular Medicine | en_US |
| dc.date.accessioned | 2022-08-04T08:09:49Z | |
| dc.date.available | 2022-08-04T08:09:49Z | |
| dc.date.issued | 2021-05-01 | en_US |
| dc.description.abstract | Restoration of correct splicing of βIVS2-654-globin pre-mRNA was previously accomplished in erythroid cells from β-thalassemia/HbE patients by an engineered U7 small nuclear RNA (snRNA) that carried a sequence targeted to the cryptic branch point and an exonic splicing enhancer, U7.BP+623 snRNA. In this study, this approach was tested in thalassemic mice carrying the βIVS2-654 mutation. While correction of βIVS2-654 pre-mRNA splicing was achieved in erythroid progenitors transduced with a lentiviral vector carrying the U7.BP+623 snRNA, a high level of truncated U7.BP+623 snRNA was also observed. The discrepancy of processing of the modified U7 snRNA in human and mouse constructs hamper the evaluation of pathologic improvement in mouse model. | en_US |
| dc.identifier.citation | Human Gene Therapy. Vol.32, No.9-10 (2021), 473-480 | en_US |
| dc.identifier.doi | 10.1089/hum.2020.145 | en_US |
| dc.identifier.issn | 15577422 | en_US |
| dc.identifier.issn | 10430342 | en_US |
| dc.identifier.other | 2-s2.0-85101504394 | en_US |
| dc.identifier.uri | https://repository.li.mahidol.ac.th/handle/20.500.14594/76199 | |
| dc.rights | Mahidol University | en_US |
| dc.rights.holder | SCOPUS | en_US |
| dc.source.uri | https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85101504394&origin=inward | en_US |
| dc.subject | Biochemistry, Genetics and Molecular Biology | en_US |
| dc.title | Engineered U7 Small Nuclear RNA Restores Correct β-Globin Pre-mRNA Splicing in Mouse β<sup>iVS2-654</sup>-Thalassemic Erythroid Progenitor Cells | en_US |
| dc.type | Article | en_US |
| dspace.entity.type | Publication | |
| mu.datasource.scopus | https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85101504394&origin=inward | en_US |