Repair of thalassemic human beta-globin mRNA in mammalian cells by antisense oligonucleotides (Q36684554)

15 August 2017

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

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15 August 2017

Sierakowska H

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15 August 2017

Sambade MJ

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15 August 2017

Agrawal S

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15 August 2017

Kole R

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15 August 2017

language of work or name

English

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publication date

1 November 1996

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

15 August 2017

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15 August 2017

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15 August 2017

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15 August 2017

12840-12844

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

15 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=24007

Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=24007

Identification and characterization by antisense oligonucleotides of exon and intron sequences required for splicing

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=24007

30 September 2017

Does antisense exist?

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=24007

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

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=24007

RNase H cleavage of RNA hybridized to oligonucleotides containing methylphosphonate, phosphorothioate and phosphodiester bonds

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=24007

Tricine-sodium dodecyl sulfate-polyacrylamide gel electrophoresis for the separation of proteins in the range from 1 to 100 kDa

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=24007

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

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=24007

Selection of splice sites in pre-mRNAs with short internal exons

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=24007

Biochemical Mechanisms of Constitutive and Regulated Pre-mRNA Splicing

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=24007

The role of hemin in the regulation of protein synthesis in erythroid cells

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=24007

Cationic lipids enhance cellular uptake and activity of phosphorothioate antisense oligonucleotides

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=24007

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

28 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=24007

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

5 September 2018

1 reference

Crossref

https://api.crossref.org/works/10.1073%2FPNAS.93.23.12840

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

1 reference

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

1 reference

12 December 2020

Clinical and laboratory effects of long-term administration of hydroxyurea to patients with sickle-cell/beta-thalassaemia

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12 December 2020

Hemin-induced acceleration of hemoglobin production in immature cultured erythroid cells: preferential enhancement of fetal hemoglobin

1 reference

12 December 2020

Extended therapy with intravenous arginine butyrate in patients with beta-hemoglobinopathies

1 reference

12 December 2020

In vivo stability, disposition and metabolism of a “hybrid” oligonucleotide phosphorothioate in rats

1 reference

12 December 2020

Identifiers

DOI

10.1073/PNAS.93.23.12840

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15 August 2017

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15 August 2017

PubMed publication ID

8917506

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