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

doi:10.1080/07328319708006154

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 journal › Article › peer-review

11 Scopus citations

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 language	English (US)
Pages (from-to)	1173-1182
Number of pages	10
Journal	Nucleosides and Nucleotides
Volume	16
Issue number	7-9
DOIs	https://doi.org/10.1080/07328319708006154
State	Published - 1997
Externally published	Yes

ASJC Scopus subject areas

Biochemistry
Genetics

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10.1080/07328319708006154

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

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.",

author = "Halina Sierakowska and Michael Montague and Sudhir Agrawal and Ryszard Kole",

year = "1997",

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AU - Sierakowska, Halina

AU - Montague, Michael

AU - Agrawal, Sudhir

AU - Kole, Ryszard

PY - 1997

Y1 - 1997

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AB - 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.

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ER -

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

Abstract

ASJC Scopus subject areas

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