Restoration of ß-Globin Gene Expression in Mammalian Cells by Antisense Oligonucleotides That Modify the Aberrant Splicing Patierns of Thalassemic Pre-mRNAs
- 1 July 1997
- journal article
- Published by Taylor & Francis in Nucleosides and Nucleotides
- Vol. 16 (7), 1173-1182
- https://doi.org/10.1080/07328319708006154
Abstract
Antisense 2′-methoxy-oligoribonucleotides targeted to aberrant splice sites in two thalassemic human ß-globin pre-mRNAs, IVS2-654 and IVS2-705, expressed in HeLa cells efficiently restore correct splicing with the use of Lipofectamine, Cellfectin and DMRIE-C, in effect reactivating a defective ß-globin gene.Keywords
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