(Q37079038)

18 August 2017

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice (English)

1 reference

18 August 2017

7

Oliver Smithies

1 reference

18 August 2017

8

Ryszard Kole

1 reference

18 August 2017

Saovaros Svasti

1

1 reference

18 August 2017

Thipparat Suwanmanee

2

1 reference

18 August 2017

Suthat Fucharoen

3

1 reference

18 August 2017

Hong M Moulton

4

1 reference

18 August 2017

Michelle H Nelson

5

1 reference

18 August 2017

Nobuyo Maeda

6

1 reference

18 August 2017

language of work or name

English

0 references

publication date

21 January 2009

1 reference

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

18 August 2017

1 reference

18 August 2017

106

1 reference

18 August 2017

1205-1210

1 reference

18 August 2017

4

1 reference

describes a project that uses

18 August 2017

ImageQuant

1 reference

11 June 2022

https://www.ebi.ac.uk/europepmc/webservices/rest/PMC2633555/fullTextXML

inferred from PubMed Central ID database lookup

cites work

Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

An endogenous TNF-alpha antagonist induced by splice-switching oligonucleotides reduces inflammation in hepatitis and arthritis mouse models

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

Correction of ClC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

Repair of a splicing defect in erythroid cells from patients with beta-thalassemia/HbE disorder

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

Control of alternative splicing by antisense oligonucleotides as a potential chemotherapy: effects on gene expression

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

Nuclear antisense effects of neutral, anionic and cationic oligonucleotide analogs

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

Sensitivity of splice sites to antisense oligonucleotides in vivo.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

Up-regulation of luciferase gene expression with antisense oligonucleotides: implications and applications in functional assay development

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

A common human beta globin splicing mutation modeled in mice.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

Systemically delivered antisense oligomers upregulate gene expression in mouse tissues

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

Hemoglobinopathies in Southeast Asia: molecular biology and clinical medicine

27 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

27 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing

4 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

HIV Tat peptide enhances cellular delivery of antisense morpholino oligomers.

4 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

Comparison of mechanisms of anemia in mice with sickle cell disease and beta-thalassemia: peripheral destruction, ineffective erythropoiesis, and phospholipid scramblase-mediated phosphatidylserine exposure

4 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2633555

Bone marrow transplantation in adult thalassemic patients

4 September 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/19164558

12 December 2020

inferred from PubMed ID database lookup

Efficient and persistent splice switching by systemically delivered LNA oligonucleotides in mice

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/19164558

12 December 2020

inferred from PubMed ID database lookup

An Endogenous TNF-α Antagonist Induced by Splice-switching Oligonucleotides Reduces Inflammation in Hepatitis and Arthritis Mouse Models

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/19164558

12 December 2020

inferred from PubMed ID database lookup

Identifiers

DOI

10.1073/PNAS.0812436106

1 reference

18 August 2017

0 references

1 reference

18 August 2017

1 reference