ArticleProceedings of the National Academy of Sciences of the United States of America1996
Repair of thalassemic human beta-globin mRNA in mammalian cells by antisense oligonucleotides.
Article in Proceedings of the National Academy of Sciences of the United States of America, 1996. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 78 papers.
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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
78 citing papers in PubMed, 237 citations in OpenAlex.
- From a Concept on a Chalkboard to Gapmer Antisense.ACS omega · 2026Article
- TDP-43: [GU]-ardian of the transcriptome.Molecular neurodegeneration · 2026Review
- Using aptamers for targeted delivery of RNA therapies.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Nucleic acid therapeutics: Past, present, and future.Molecular therapy. Nucleic acids · 2025Review
- ASOG: AntiSense Oligonucleotide Generator.Computational and structural biotechnology journal · 2025Article
- Evolution and Breakthroughs in Exon Skipping and Splice Modulation: From Inception to Clinical Success.Methods in molecular biology (Clifton, N.J.) · 2025Article
- Morpholino oligonucleotide-mediated exon skipping for DMD treatment: Past insights, present challenges and future perspectives.Journal of biosciences · 2023Review
- Thiomorpholino oligonucleotides as a robust class of next generation platforms for alternate mRNA splicing.Proceedings of the National Academy of Sciences of the United States of America · 2022Article
- An LNA-amide modification that enhances the cell uptake and activity of phosphorothioate exon-skipping oligonucleotides.Nature communications · 2022Article
- Covalently attached intercalators restore duplex stability and splice-switching activity to triazole-modified oligonucleotides.RSC chemical biology · 2022Article
- Introduction and History of the Chemistry of Nucleic Acids Therapeutics.Methods in molecular biology (Clifton, N.J.) · 2022Article
- Antisense Oligonucleotide-Mediated Splice Switching: Potential Therapeutic Approach for Cancer Mitigation.Cancers · 2021Review
- Delivery of Oligonucleotide Therapeutics: Chemical Modifications, Lipid Nanoparticles, and Extracellular Vesicles.ACS nano · 2021Review
- The Evolution of Antisense Oligonucleotide Chemistry-A Personal Journey.Biomedicines · 2021Review
- Antisense oligonucleotide technology can be used to investigate a circular but not linear RNA-mediated function for its encoded gene locus.Science China. Life sciences · 2021Article
- Evaluation of DNA segments in 2'-modified RNA sequences in designing efficient splice switching antisense oligonucleotides.RSC advances · 2021Article
- U7 snRNA: A tool for gene therapy.The journal of gene medicine · 2021Review
- Article
- RNA Therapeutics Are Stepping Out of the Maze.Trends in molecular medicine · 2020Review
- Restoration of correct βScientific reports · 2019Article
18 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 1 institution.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In one form of beta-thalassemia, a genetic blood disorder, a mutation in intron 2 of the beta-globin gene (IVS2-654) causes aberrant splicing of beta-globin pre-mRNA and, consequently, beta-globin deficiency. Treatment of mammalian cells stably expressing the IVS2-654 human beta-globin gene with antisense oligonucleotides targeted at the aberrant splice sites restored correct splicing in a dose-dependent fashion, generating correct human beta-globin mRNA and polypeptide. Both products persisted for up to 72 hr posttreatment. The oligonucleotides modified splicing by a true antisense mechanism without overt unspecific effects on cell growth and splicing of other pre-mRNAs. This novel approach in which antisense oligonucleotides are used to restore rather than to down-regulate the activity of the target gene is applicable to other splicing mutants and is of potential clinical interest.
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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.