ArticleEMBO molecular medicine2013
Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds.
Article in EMBO molecular medicine, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 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
27 citing papers in PubMed, 46 citations in OpenAlex.
- The effect of coadministration of D156844 and AR42 (REC-2282) on the survival and motor phenotype of mice with spinal muscular atrophy.Scientific reports · 2025Article
- Understanding crosstalk of organ tropism, tumor microenvironment and noncoding RNAs in breast cancer metastasis.Molecular biology reports · 2023Review
- Repurposing FDA-approved drugs as inhibitors of therapy-induced invadopodia activity in glioblastoma cells.Molecular and cellular biochemistry · 2023Article
- History of development of the life-saving drug "Nusinersen" in spinal muscular atrophy.Frontiers in cellular neuroscience · 2022Review
- Short-duration splice promoting compound enables a tunable mouse model of spinal muscular atrophy.Life science alliance · 2021Article
- Small molecule targeting of RNA structures in neurological disorders.Annals of the New York Academy of Sciences · 2020Review
- ZPR1 prevents R-loop accumulation, upregulates SMN2 expression and rescues spinal muscular atrophy.Brain : a journal of neurology · 2020Article
- Drug Screening and Drug Repositioning as Promising Therapeutic Approaches for Spinal Muscular Atrophy Treatment.Frontiers in pharmacology · 2020Review
- Intraperitoneal delivery of a novel drug-like compound improves disease severity in severe and intermediate mouse models of Spinal Muscular Atrophy.Scientific reports · 2019Article
- Optimization of a series of heterocycles as survival motor neuron gene transcription enhancers.Bioorganic & medicinal chemistry letters · 2017Article
- Ventromorphins: A New Class of Small Molecule Activators of the Canonical BMP Signaling Pathway.ACS chemical biology · 2017Article
- Discovery of a Small Molecule Probe That Post-Translationally Stabilizes the Survival Motor Neuron Protein for the Treatment of Spinal Muscular Atrophy.Journal of medicinal chemistry · 2017Article
- Article
- Small Molecules in Development for the Treatment of Spinal Muscular Atrophy.Journal of medicinal chemistry · 2016Review
- Protective effects of butyrate-based compounds on a mouse model for spinal muscular atrophy.Experimental neurology · 2016Article
- CNS uptake of bortezomib is enhanced by P-glycoprotein inhibition: implications for spinal muscular atrophy.Neurobiology of disease · 2016Article
- Molecular Mechanisms of Neurodegeneration in Spinal Muscular Atrophy.Journal of experimental neuroscience · 2016Review
- RNA mis-splicing in disease.Nature reviews. Genetics · 2016Review
- Genetic inhibition of JNK3 ameliorates spinal muscular atrophy.Human molecular genetics · 2015Article
- The effect of the DcpS inhibitor D156844 on the protective action of follistatin in mice with spinal muscular atrophy.Neuromuscular disorders : NMD · 2015Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 6 institutions in 1 country.
Funding
Abstract
Spinal muscular atrophy (SMA) is a neurodegenerative disease that causes progressive muscle weakness, which primarily targets proximal muscles. About 95% of SMA cases are caused by the loss of both copies of the SMN1 gene. SMN2 is a nearly identical copy of SMN1, which expresses much less functional SMN protein. SMN2 is unable to fully compensate for the loss of SMN1 in motor neurons but does provide an excellent target for therapeutic intervention. Increased expression of functional full-length SMN protein from the endogenous SMN2 gene should lessen disease severity. We have developed and implemented a new high-throughput screening assay to identify small molecules that increase the expression of full-length SMN from a SMN2 reporter gene. Here, we characterize two novel compounds that increased SMN protein levels in both reporter cells and SMA fibroblasts and show that one increases lifespan, motor function, and SMN protein levels in a severe mouse model of SMA.
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.