ReviewCurrent opinion in molecular therapeutics2009
Splicing therapeutics in SMN2 and APOB.
Review in Current opinion in molecular therapeutics, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
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
18 citing papers in PubMed, 1 synthesis or guideline pooled it, 27 citations in OpenAlex.
- Multiancestry Genome-Wide Association Study of Lipid Levels Incorporating Gene-Alcohol Interactions.American journal of epidemiology · 2019Pooled it
- Control of epithelial tissue organization by mRNA localization.Nature communications · 2025Article
- In vivo PAR-CLIP (viP-CLIP) of liver TIAL1 unveils targets regulating cholesterol synthesis and secretion.Nature communications · 2023Article
- [Molecular medicine: pathobiochemistry as the key to personalized treatment of inherited diseases].Monatsschrift Kinderheilkunde : Organ der Deutschen Gesellschaft fur Kinderheilkunde · 2021Review
- Hepatic HuR modulates lipid homeostasis in response to high-fat diet.Nature communications · 2020Article
- Article
- Mechanisms of Neuronal Alternative Splicing and Strategies for Therapeutic Interventions.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2019Review
- Oligonucleotide Therapies: The Past and the Present.Human gene therapy · 2015Review
- Integrative genome-wide analysis of the determinants of RNA splicing in kidney renal clear cell carcinoma.Pacific Symposium on Biocomputing. Pacific Symposium on Biocomputing · 2015Article
- Development of therapeutic splice-switching oligonucleotides.Human gene therapy · 2014Review
- Antisense therapy in neurology.Journal of personalized medicine · 2013Review
- The little elongation complex regulates small nuclear RNA transcription.Molecular cell · 2011Article
- Regulation of the mutually exclusive exons 8a and 8 in the CaV1.2 calcium channel transcript by polypyrimidine tract-binding protein.The Journal of biological chemistry · 2011Article
- Developments in RNA splicing and disease.Cold Spring Harbor perspectives in biology · 2011Review
- Impairment of pre-mRNA splicing in liver disease: mechanisms and consequences.World journal of gastroenterology · 2010Review
- Therapy development in spinal muscular atrophy.Nature neuroscience · 2010Article
- Spinal muscular atrophy: mechanisms and therapeutic strategies.Human molecular genetics · 2010Review
- Therapeutic RNA manipulation in liver disease.Hepatology (Baltimore, Md.) · 2010Review
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 at 1 institution in 1 country.
Funding
Abstract
Splicing therapeutics are defined as the deliberate modification of RNA splicing to achieve therapeutic goals. Various techniques for splicing therapeutics have been described, and most of these involve the use of antisense oligonucleotide-based compounds that target key elements in the pre-mRNA to control splicing in the nucleus. In this review, recent developments in splicing therapeutics for the treatment of two specific diseases are described: correcting the alternative splicing of survival of motor neuron (SMN)2 pre-mRNA to compensate for the defective SMN1 gene in spinal muscular atrophy, and re-engineering the splicing of apolipoprotein B pre-mRNA to lower circulating cholesterol levels.
Indexed as
Identifiers
19330716PMC3140428W1807973539What 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.