ReviewMethods in molecular biology (Clifton, N.J.)2025
Progress and Future Directions in Exon Skipping and Inclusion Therapies: The Landscape of Oligonucleotide-Based Genetic Medicine.
Review in Methods in molecular biology (Clifton, N.J.), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
The trial behind it
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
2 citing papers in PubMed.
- Review
- A therapeutic antisense oligonucleotide encompassing 2'-NAR molecular medicine · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Oligonucleotide (ON)-based therapies, particularly exon skipping and inclusion strategies, have revolutionized the treatment landscape for genetic and rare diseases, heralding a new era in personalized medicine. This chapter traces the evolution of ON therapeutics, from pioneering efforts in the 1970s to their resurgence as cutting-edge treatments in the twenty-first century. Highlighting seminal developments, such as the approval of splice-switching ONs like eteplirsen for Duchenne Muscular Dystrophy and nusinersen for Spinal Muscular Atrophy, it underscores the critical role of these therapies in advancing patient-specific treatments. Despite their promising potential, exon skipping and inclusion therapies encounter challenges related to delivery, stability, and regulatory hurdles. Commemorating the silver jubilee of the first ON therapy approval, this narrative not only celebrates the achievements but also critically examines the ongoing challenges and the imperative for sustained innovation and collaboration. The chapter aims to illuminate the path forward, emphasizing the need to harness the full promise of exon skipping and inclusion therapies in personalized genetic medicine.
Indexed as
Identifiers
40720008What 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.