Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides.
about
New approaches to the treatment of orphan genetic disorders: Mitigating molecular pathologies using chemicalsSilencing disease genes in the laboratory and the clinicModulation of mdm2 pre-mRNA splicing by 9-aminoacridine-PNA (peptide nucleic acid) conjugates targeting intron-exon junctions.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.Correction of aberrant FGFR1 alternative RNA splicing through targeting of intronic regulatory elements.Splice-correction strategies for treatment of X-linked agammaglobulinemia.Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing.Correction of prototypic ATM splicing mutations and aberrant ATM function with antisense morpholino oligonucleotides.Small regulatory RNAs in mammals.Modulation of gene expression by antisense and antigene oligodeoxynucleotides and small interfering RNA.Molecular therapies in beta-thalassaemia.Nuclear antisense effects in cyclophilin A pre-mRNA splicing by oligonucleotides: a comparison of tricyclo-DNA with LNARNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.Therapeutic potential of splice-switching oligonucleotides.Antisense oligonucleotide-based therapy in human erythropoietic protoporphyria.Alternatively spliced mu opioid receptor C termini impact the diverse actions of morphine.Gene therapy in thalassemia and hemoglobinopathies.Recent advances in gene therapy for thalassemia.Temperature-dependent splicing of beta-globin pre-mRNA.Antisense therapeutics for neurofibromatosis type 1 caused by deep intronic mutations.Generation and Characterization of a Transgenic Mouse Carrying a Functional Human β -Globin Gene with the IVSI-6 Thalassemia Mutation.Restoration of correct splicing in IVSI-110 mutation of β-globin gene with antisense oligonucleotides: implications and applications in functional assay development.Sodium Butyrate and Valproic Acid as Splicing Restoring Agents in Erythroid Cells of β-Thalassemic Patients.Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia.Alternative Splicing as a Target for Cancer Treatment.
P2860
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P2860
Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides.
description
2002 nî lūn-bûn
@nan
2002 թուականի Սեպտեմբերին հրատարակուած գիտական յօդուած
@hyw
2002 թվականի սեպտեմբերին հրատարակված գիտական հոդված
@hy
2002年の論文
@ja
2002年論文
@yue
2002年論文
@zh-hant
2002年論文
@zh-hk
2002年論文
@zh-mo
2002年論文
@zh-tw
2002年论文
@wuu
name
Restoration of human beta-glob ...... of antisense oligonucleotides.
@ast
Restoration of human beta-glob ...... of antisense oligonucleotides.
@en
Restoration of human beta-glob ...... of antisense oligonucleotides.
@nl
type
label
Restoration of human beta-glob ...... of antisense oligonucleotides.
@ast
Restoration of human beta-glob ...... of antisense oligonucleotides.
@en
Restoration of human beta-glob ...... of antisense oligonucleotides.
@nl
prefLabel
Restoration of human beta-glob ...... of antisense oligonucleotides.
@ast
Restoration of human beta-glob ...... of antisense oligonucleotides.
@en
Restoration of human beta-glob ...... of antisense oligonucleotides.
@nl
P2093
P356
P1476
Restoration of human beta-glob ...... of antisense oligonucleotides
@en
P2093
Christopher E Walsh
Giuseppina Lacerra
Halina Sierakowska
Suthat Fucharoen
Suzanne Kirby
Thipparat Suwanmanee
P304
P356
10.1124/MOL.62.3.545
P577
2002-09-01T00:00:00Z