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

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

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

http://www.wikidata.org/entity/Q36684554

about

Nuclear antisense effects of neutral, anionic and cationic oligonucleotide analogs Antisense morpholino-oligomers directed against the 5' end of the genome inhibit coronavirus proliferation and growth Stable alteration of pre-mRNA splicing patterns by modified U7 small nuclear RNAs Correction of a splice-site mutation in the beta-globin gene stimulated by triplex-forming peptide nucleic acids Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications Doxycycline-controlled splicing modulation by regulated antisense U7 snRNA expression cassettes Oligonucleotides and derivatives as gene-specific control agents.Antisense properties of tricyclo-DNA Antisense oligonucleotide-induced alternative splicing of the APOB mRNA generates a novel isoform of APOB.Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon.Targeted correction of a thalassemia-associated beta-globin mutation induced by pseudo-complementary peptide nucleic acids Future alternative therapies for β-thalassemia.MBNL1 binds GC motifs embedded in pyrimidines to regulate alternative splicing.Design and evaluation of locked nucleic acid-based splice-switching oligonucleotides in vitro.Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cells The potential of nucleic acid repair in functional genomics.Potential therapeutic application of antisense oligonucleotides in the treatment of ocular diseases.Sensitivity of splice sites to antisense oligonucleotides in vivo.Anti-tumor activity of splice-switching oligonucleotides.Human atopic dermatitis complicated by eczema herpeticum is associated with abnormalities in IFN-γ response.The regulation of splice-site selection, and its role in human disease Therapeutic potential of antisense nucleic acid molecules.Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides.Genetic variants in interferon regulatory factor 2 (IRF2) are associated with atopic dermatitis and eczema herpeticum.mRNA transcript diversity creates new opportunities for pharmacological intervention.Epigenetic manipulation of gene expression: a toolkit for cell biologists Molecular therapies in beta-thalassaemia.Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice.Directing HER4 mRNA expression towards the CYT2 isoform by antisense oligonucleotide decreases growth of breast cancer cells in vitro and in vivo.Therapeutic applications of aptamers.Delineation of the mechanisms of aberrant splicing caused by two unusual intronic mutations in the RSK2 gene involved in Coffin-Lowry syndrome.RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.Modification of HER2 pre-mRNA alternative splicing and its effects on breast cancer cells.Advances in antisense oligonucleotide development for target identification, validation, and as novel therapeutics.Therapeutic potential of splice-switching oligonucleotides.Neuronal ceroid lipofuscinosis type CLN2: a new rationale for the construction of phenotypic subgroups based on a survey of 25 cases in South America.Alternative splicing: role of pseudoexons in human disease and potential therapeutic strategies.Gene therapy in thalassemia and hemoglobinopathies.

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P2860

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

http://www.wikidata.org/entity/Q36684554

description

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name

Repair of thalassemic human be ...... by antisense oligonucleotides

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Repair of thalassemic human be ...... by antisense oligonucleotides

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Repair of thalassemic human be ...... by antisense oligonucleotides

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Repair of thalassemic human be ...... by antisense oligonucleotides

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P2093

Agrawal S

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

Kole R

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Sambade MJ

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Sierakowska H

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P2860

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

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

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

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

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

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

Biochemical mechanisms of constitutive and regulated pre-mRNA splicing.

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

Does antisense exist?

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

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