ReviewPLoS genetics2007
Modulating the expression of disease genes with RNA-based therapy.
Review in PLoS genetics, 2007. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 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
47 citing papers in PubMed, 96 citations in OpenAlex.
- Review
- Transforming RNA-Based Gene Therapy with Innovative Nanocarriers for siRNA and miRNA Delivery.Current gene therapy · 2026Review
- Introns and Their Therapeutic Applications in Biomedical Researches.Iranian journal of biotechnology · 2023Review
- Antisense Oligonucleotide Treatment in a Humanized Mouse Model of Duchenne Muscular Dystrophy and Highly Sensitive Detection of Dystrophin Using Western Blotting.Methods in molecular biology (Clifton, N.J.) · 2021Article
- Use of Nanoparticles in Delivery of Nucleic Acids for Melanoma Treatment.Methods in molecular biology (Clifton, N.J.) · 2021Article
- Gene therapy for inherited arrhythmias.Cardiovascular research · 2020Review
- Loci specific epigenetic drug sensitivity.Nucleic acids research · 2020Article
- Gene Therapy via Trans-Splicing for LMNA-Related Congenital Muscular Dystrophy.Molecular therapy. Nucleic acids · 2018Article
- Impact, Characterization, and Rescue of Pre-mRNA Splicing Mutations in Lysosomal Storage Disorders.Genes · 2018Review
- Trans-splicing repair of mutant p53 suppresses the growth of hepatocellular carcinoma cells in vitro and in vivo.Scientific reports · 2015Article
- Article
- The analytical landscape of static and temporal dynamics in transcriptome data.Frontiers in genetics · 2014Review
- An emerging role for long non-coding RNA dysregulation in neurological disorders.International journal of molecular sciences · 2013Review
- Multiple tandem splicing silencer elements suppress aberrant splicing within the long exon 26 of the human Apolipoprotein B gene.BMC molecular biology · 2013Article
- Emerging molecular targets for brain repair after stroke.Stroke research and treatment · 2013Article
- Antisense-induced messenger depletion corrects a COL6A2 dominant mutation in Ullrich myopathy.Human gene therapy · 2012Article
- Replacement of huntingtin exon 1 by trans-splicing.Cellular and molecular life sciences : CMLS · 2012Article
- Illuminating the gateway of gene silencing: perspective of RNA interference technology in clinical therapeutics.Molecular biotechnology · 2012Review
- Alu-Alu Recombination Underlying the First Large Genomic Deletion in GlcNAc-Phosphotransferase Alpha/Beta (GNPTAB) Gene in a MLII Alpha/Beta Patient.JIMD reports · 2012Article
- The Emerging Role of Non-Coding RNAs in Drug Addiction.Frontiers in genetics · 2012Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 1 country.
Funding
Abstract
Conventional gene therapy has focused largely on gene replacement in target cells. However, progress from basic research to the clinic has been slow for reasons relating principally to the challenges of heterologous DNA delivery and regulation in vivo. Alternative approaches targeting RNA have the potential to circumvent some of these difficulties, particularly as the active therapeutic molecules are usually short oligonucleotides and the target gene transcript is under endogenous regulation. RNA-based strategies offer a series of novel therapeutic applications, including altered processing of the target pre-mRNA transcript, reprogramming of genetic defects through mRNA repair, and the targeted silencing of allele- or isoform-specific gene transcripts. This review examines the potential of RNA therapeutics, focusing on antisense oligonucleotide modification of pre-mRNA splicing, methods for pre-mRNA trans-splicing, and the isoform- and allele-specific applications of RNA interference.
Indexed as
Identifiers
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.