ReviewInternational journal of molecular sciences2025
Expansion of Splice-Switching Therapy with Antisense Oligonucleotides.
Review in International journal of molecular sciences, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
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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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Who cites it
8 citing papers in PubMed.
- Relevance of functional studies for assessing an antisense oligonucleotide-mediated exon skipping therapeutic strategy for mucolipidosis type II.Orphanet journal of rare diseases · 2026Article
- Correction of aberrant splicing caused by intronic CAPN3 pathogenic variants using RNA-targeted therapeutic strategies in limb-girdle muscular dystrophy type R1.Orphanet journal of rare diseases · 2026Article
- Homozygous SGCB splice-site variant causes isolated dilated cardiomyopathy through sarcoglycan complex destabilization in East Asians.The Journal of clinical investigation · 2026Article
- Nucleic-Acid-Based Strategies and Nanotechnology Applications for Targeted Therapy.Pharmaceutics · 2026Article
- Unconventional Lysine-Type Lipid Assemblies Enable Efficient Antisense Oligonucleotide Delivery with Distinct Structural Features.Pharmaceutics · 2026Article
- Unlocking the undruggable spliceosome: generative AI and structural dynamics in cancer therapy.Frontiers in cell and developmental biology · 2026Review
- Article
- A New High Penetrant Intronic Pathogenic Variant Related to Long QT Syndrome Type 2.Journal of clinical medicine · 2025Article
Corrections and comments
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Authors and funding
1 author.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Since 2016, splice-switching therapy, in which splicing is controlled by antisense oligonucleotides, has been applied in clinical practice for spinal muscular atrophy and Duchenne muscular dystrophy. In the former disease, this therapy induces exon inclusion, while, in the latter, it induces exon skipping, leading expression of functional proteins. Basic and clinical studies of splice-switching therapy for many monogenic diseases have now been conducted. The molecular mechanisms of splice-switching therapy include not only the induction of exon inclusion and skipping, but also the induction of pseudoexon skipping and suppression of splicing sites generated by mutations. In addition, therapies that alter protein function by regulating splicing are being investigated not only for monogenic diseases but also for non-monogenic ones such as cancer and immune-related disorders. It is expected that many of these basic studies will be translated into clinical applications. This review describes the current status of basic research and clinical applications of splice-switching therapy to promote the development of treatments for noncurable diseases.
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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.