(Q24670228)

2

0 references

C Carestia

3

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S Fucharoen

4

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J Summerton

5

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D Weller

6

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R Kole

Proceedings of the National Academy of Sciences of the United States of America

7

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language of work or name

English

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publication date

15 August 2000

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published in

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volume

97

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page(s)

9591-6

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issue

17

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cites work

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7 April 2017

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Direct selection for mutations affecting specific splice sites in a hamster dihydrofolate reductase minigene

7 April 2017

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Clinical and hematologic aspects of hemoglobin E beta-thalassemia

7 April 2017

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Antisense therapeutics: is it as simple as complementary base recognition?

28 September 2017

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Preclinical and clinical pharmacology of antisense oligonucleotides

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Induction of endogenous Bcl-xS through the control of Bcl-x pre-mRNA splicing by antisense oligonucleotides

28 September 2017

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Antisense oligonucleotides with different backbones. Modification of splicing pathways and efficacy of uptake

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Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides.

28 September 2017

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The beta-thalassemias

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Sensitivity of splice sites to antisense oligonucleotides in vivo.

28 September 2017

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Development of viral vectors for gene therapy of beta-chain hemoglobinopathies: optimization of a gamma-globin gene expression cassette

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Reversal of lethal alpha- and beta-thalassemias in mice by expression of human embryonic globins

28 September 2017

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28 September 2017

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The ups and downs of nucleic acid duplex stability: structure-stability studies on chemically-modified DNA:RNA duplexes

28 September 2017

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Repair of thalassemic human beta-globin mRNA in mammalian cells by antisense oligonucleotides

28 September 2017

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A simple method for delivering morpholino antisense oligos into the cytoplasm of cells

28 September 2017

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beta-Thalassemia in Chinese: use of in vivo RNA analysis and oligonucleotide hybridization in systematic characterization of molecular defects

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Frequencies of common beta-thalassaemia alleles among different populations: variability in clinical severity

28 September 2017

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Correction of aberrant splicing of the cystic fibrosis transmembrane conductance regulator (CFTR) gene by antisense oligonucleotides

28 September 2017

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A single-base change at a splice site in a beta 0-thalassemic gene causes abnormal RNA splicing.

30 May 2018

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Beta + thalassemia: aberrant splicing results from a single point mutation in an intron.

30 May 2018

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Crossref

https://api.crossref.org/works/10.1073%2FPNAS.97.17.9591

Specific transcription and RNA splicing defects in five cloned β-thalassaemia genes

21 January 2018

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https://api.crossref.org/works/10.1073%2FPNAS.97.17.9591

Abnormally spliced messenger RNA in erythroid cells from patients with β+ thalassemia and monkey cells expressing a cloned β+-thalassemic gene

21 January 2018

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12 December 2020

Reversibility of IVS 2 missplicing in a mutant human beta-globin gene

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12 December 2020

RNA transcripts of the beta-thalassaemia allele IVS-2-654 C-->T: a small amount of normally processed beta-globin mRNA is still produced from the mutant gene

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12 December 2020

Isobutyramide, an orally bioavailable butyrate analogue, stimulates fetal globin gene expression in vitro and in vivo

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12 December 2020

The therapeutic reactivation of fetal haemoglobin

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12 December 2020

PAMAM dendrimers as delivery agents for antisense oligonucleotides

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12 December 2020