ArticleIranian journal of biotechnology2016
Sodium Butyrate and Valproic Acid as Splicing Restoring Agents in Erythroid Cells of β-Thalassemic Patients.
Article in Iranian journal of biotechnology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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1 citing paper in PubMed, 0 citations in OpenAlex.
- Microbiota-derived metabolites in hematopoietic regulation and disease: mechanisms, context, and therapeutic perspectives.Frontiers in microbiology · 2026Review
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4 authors at 1 institution in 1 country.
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No grant is acknowledged in the PubMed record.
Abstract
backgroundβ-Thalassemia is a common autosomal recessive disorder in human caused by a defect in
objectivesThe aim of this study was to investigate the effects of NaBu, isoBu and VPA drugs on restoration of splicing of IVSI-110 β-Thalassemia pre-mRNA in human. MATERIALS AND
methodsPrimary erythroid cells derived from IVSI-110 β-Thalassemia patients were cultured ex vivo and differentiated in the presence of 0.5 and 1 mM of Na-Butyrate (NaBu), 0.5 mM Isobutyramide (isoBu) and 100 μM Valproic acid (VPA). RT- PCR analysis was used to evaluate the effect of the drugs in correction of normal splicing in bglobin mRNAs.
resultsFollowing treatment with NaBu, isoBu and VPA, the level of normal
conclusionsThe results highlighted the therapeutic potential of splicing modulation for genetic diseases caused by splicing mutations.
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