Restoration of β-globin gene expression in mammalian cells by antisense oligonucleotides that modify the aberrant splicing patterns of thalassemic pre-mRNAs

Halina Sierakowska, Michael Montague, Sudhir Agrawal, Ryszard Kole

Research output: Contribution to journalArticle

Abstract

Antisense 2'-methoxy-oligoribonucleotides targeted to aberrant splice sites in two thalassemic human β-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 β- globin gene.

Original languageEnglish (US)
Pages (from-to)1173-1182
Number of pages10
JournalNucleosides and Nucleotides
Volume16
Issue number7-9
DOIs
StatePublished - Jan 1 1997
Externally publishedYes

ASJC Scopus subject areas

  • Biochemistry
  • Genetics

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