RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice. - Wikidata - wikimedia - TriplyDB

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.

http://www.wikidata.org/entity/Q37079038

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RNA-Binding Proteins: Splicing Factors and Disease Animal models of β-hemoglobinopathies: utility and limitations RNA therapeutics: RNAi and antisense mechanisms and clinical applications In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery Future alternative therapies for β-thalassemia.Enhancement of β-Globin Gene Expression in Thalassemic IVS2-654 Induced Pluripotent Stem Cell-Derived Erythroid Cells by Modified U7 snRNA.Gene knockdowns in adult animals: PPMOs and vivo-morpholinos.Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model RNA therapeutics: beyond RNA interference and antisense oligonucleotides.Anti-tumor activity of splice-switching oligonucleotides.Efficient in vivo manipulation of alternative pre-mRNA splicing events using antisense morpholinos in mice.Regulation of the mutually exclusive exons 8a and 8 in the CaV1.2 calcium channel transcript by polypyrimidine tract-binding protein.Antisense Modulation of RNA Processing as a Therapeutic Approach in Cancer Therapy.Splice-correction strategies for treatment of X-linked agammaglobulinemia.Targeted gene modification of hematopoietic progenitor cells in mice following systemic administration of a PNA-peptide conjugate Molecular target validation, antimicrobial delivery, and potential treatment of Toxoplasma gondii infections Targeting RNA splicing for disease therapy.Directing HER4 mRNA expression towards the CYT2 isoform by antisense oligonucleotide decreases growth of breast cancer cells in vitro and in vivo.Splice-switching antisense oligonucleotides as therapeutic drugs Antisense oligonucleotide-based therapy in human erythropoietic protoporphyria.New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.Gene therapy in thalassemia and hemoglobinopathies.Alternative options for DNA-based experimental therapy of β-thalassemia.Cell-penetrating peptides for the delivery of nucleic acids.Recent advances in gene therapy for thalassemia.Splicing modulation therapy in the treatment of genetic diseases.Recent patents and technology transfer for molecular diagnosis of β-thalassemia and other hemoglobinopathies.Nanotechnology for delivery of peptide nucleic acids (PNAs)High-Throughput Assays to Assess the Functional Impact of Genetic Variants: A Road Towards Genomic-Driven Medicine.Modulation of RNA splicing as a potential treatment for cancer.Generation and Characterization of a Transgenic Mouse Carrying a Functional Human β -Globin Gene with the IVSI-6 Thalassemia Mutation.Novel viral vectors utilizing intron splice-switching to activate genome rescue, expression and replication in targeted cells.Alternative Splicing as a Target for Cancer Treatment.

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P2860

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.

http://www.wikidata.org/entity/Q37079038

description

article científic

@ca

article scientifique

@fr

articolo scientifico

@it

artigo científico

@pt

bilimsel makale

@tr

scientific article published on 21 January 2009

@en

vedecký článok

@sk

vetenskaplig artikel

@sv

videnskabelig artikel

@da

vědecký článek

@cs

name

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.

@en

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.

@nl

type

label

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.

@en

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.

@nl

prefLabel

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.

@en

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.

@nl

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PNAS.0812436106

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

P1476

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.

@en

P2093

Hong M Moulton

http://www.w3.org/2001/XMLSchema#string

Michelle H Nelson

http://www.w3.org/2001/XMLSchema#string

Nobuyo Maeda

http://www.w3.org/2001/XMLSchema#string

Saovaros Svasti

http://www.w3.org/2001/XMLSchema#string

Suthat Fucharoen

http://www.w3.org/2001/XMLSchema#string

Thipparat Suwanmanee

http://www.w3.org/2001/XMLSchema#string

P2860

Nuclear antisense effects of neutral, anionic and cationic oligonucleotide analogs

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

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

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

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

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

Sensitivity of splice sites to antisense oligonucleotides in vivo.

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

Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing.

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

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1205-1210

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scholarly article

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10.1073/PNAS.0812436106

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Oliver Smithies

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2009-01-21T00:00:00Z

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