ArticleProceedings of the National Academy of Sciences of the United States of America2000
Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2000. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 38 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
38 citing papers in PubMed, 156 citations in OpenAlex.
- Evolution and Breakthroughs in Exon Skipping and Splice Modulation: From Inception to Clinical Success.Methods in molecular biology (Clifton, N.J.) · 2025Article
- Genetic Manipulation Strategies for β-Thalassemia: A Review.Frontiers in pediatrics · 2022Review
- Nucleic Acid Therapy for β-Thalassemia.Biologics : targets & therapy · 2020Review
- Restoration of correct βScientific reports · 2019Article
- The potential of antisense oligonucleotide therapies for inherited childhood lung diseases.Molecular and cellular pediatrics · 2018Review
- Restoration of correct splicing in IVSI-110 mutation of β-globin gene with antisense oligonucleotides: implications and applications in functional assay development.Iranian journal of basic medical sciences · 2017Article
- Enhancement of β-Globin Gene Expression in Thalassemic IVS2-654 Induced Pluripotent Stem Cell-Derived Erythroid Cells by Modified U7 snRNA.Stem cells translational medicine · 2017Article
- Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia.Advances in experimental medicine and biology · 2017Review
- Sodium Butyrate and Valproic Acid as Splicing Restoring Agents in Erythroid Cells of β-Thalassemic Patients.Iranian journal of biotechnology · 2016Article
- Approaches to Validate and Manipulate RNA Targets with Small Molecules in Cells.Annual review of pharmacology and toxicology · 2016Review
- Article
- Alternative splicing impairs soluble guanylyl cyclase function in aortic aneurysm.American journal of physiology. Heart and circulatory physiology · 2014Article
- In vitro antisense therapeutics for a deep intronic mutation causing Neurofibromatosis type 2.European journal of human genetics : EJHG · 2013Article
- AON-mediated Exon Skipping Restores Ciliation in Fibroblasts Harboring the Common Leber Congenital Amaurosis CEP290 Mutation.Molecular therapy. Nucleic acids · 2012Article
- Correction of β654-thalassaemia mice using direct intravenous injection of siRNA and antisense RNA vectors.International journal of hematology · 2011Article
- The molecular landscape of propionic acidemia and methylmalonic aciduria in Latin America.Journal of inherited metabolic disease · 2010Article
- Therapeutic RNA manipulation in liver disease.Hepatology (Baltimore, Md.) · 2010Review
- Future alternative therapies for β-thalassemia.Expert review of hematology · 2009Article
- Gene therapy in thalassemia and hemoglobinopathies.Mediterranean journal of hematology and infectious diseases · 2009Article
- Therapeutic potential of splice-switching oligonucleotides.Oligonucleotides · 2009Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 3 institutions in 3 countries.
Funding
Abstract
Mononuclear cells from peripheral blood of thalassemic patients were treated with morpholino oligonucleotides antisense to aberrant splice sites in mutant beta-globin precursor mRNAs (pre-mRNAs). The oligonucleotides restored correct splicing and translation of beta-globin mRNA, increasing the hemoglobin (Hb) A synthesis in erythroid cells from patients with IVS2-654/beta(E), IVS2-745/IVS2-745, and IVS2-745/IVS2-1 genotypes. The maximal Hb A level for repaired IVS2-745 mutation was approximately 30% of normal; Hb A was still detectable 9 days after a single treatment with oligonucleotide. Thus, expression of defective beta-globin genes was repaired and significant level of Hb A was restored in a cell population that would be targeted in clinical applications of this approach.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.