ReviewNeuroRx : the journal of the American Society for Experimental NeuroTherapeutics2006
Therapeutics development for spinal muscular atrophy.
Review in NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 42 papers.
What it found
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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.
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
42 citing papers in PubMed, 97 citations in OpenAlex.
- ERK MAPK signaling pathway inhibition as a potential target to prevent autophagy alterations in Spinal Muscular Atrophy motoneurons.Cell death discovery · 2023Article
- DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration.JCI insight · 2023Article
- Splicing efficiency of minor introns in a mouse model of SMA predominantly depends on their branchpoint sequence and can involve the contribution of major spliceosome components.RNA (New York, N.Y.) · 2022Article
- Suppression of the necroptotic cell death pathways improves survival inFrontiers in cellular neuroscience · 2022Article
- Calpain system is altered in survival motor neuron-reduced cells from in vitro and in vivo spinal muscular atrophy models.Cell death & disease · 2020Article
- Histone Deacetylases Inhibitors in Neurodegenerative Diseases, Neuroprotection and Neuronal Differentiation.Frontiers in pharmacology · 2020Review
- Calpain Inhibition Increases SMN Protein in Spinal Cord Motoneurons and Ameliorates the Spinal Muscular Atrophy Phenotype in Mice.Molecular neurobiology · 2019Article
- Regulation of Survival Motor Neuron Protein by the Nuclear Factor-Kappa B Pathway in Mouse Spinal Cord Motoneurons.Molecular neurobiology · 2018Article
- Frequency ofExperimental and therapeutic medicine · 2015Article
- Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.American journal of human genetics · 2015Article
- Investigations of curcumin and resveratrol on neurite outgrowth: perspectives on spinal muscular atrophy.BioMed research international · 2014Article
- Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds.EMBO molecular medicine · 2013Article
- Survival motor neuron protein reduction deregulates autophagy in spinal cord motoneurons in vitro.Cell death & disease · 2013Article
- Silencing human genetic diseases with oligonucleotide-based therapies.Human genetics · 2013Review
- Article
- Reliability and validity of the TIMPSI for infants with spinal muscular atrophy type I.Pediatric physical therapy : the official publication of the Section on Pediatrics of the American Physical Therapy Association · 2013Article
- Identification of novel compounds that increase SMN protein levels using an improved SMN2 reporter cell assay.Journal of biomolecular screening · 2012Article
- Identification of a Maleimide-Based Glycogen Synthase Kinase-3 (GSK-3) Inhibitor, BIP-135, that Prolongs the Median Survival Time of Δ7 SMA KO Mouse Model of Spinal Muscular Atrophy.ACS chemical neuroscience · 2012Article
- The canonical nuclear factor-κB pathway regulates cell survival in a developmental model of spinal cord motoneurons.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2011Article
- Carboxylic acid derivatives of histone deacetylase inhibitors induce full length SMN2 transcripts: a promising target for spinal muscular atrophy therapeutics.Archives of medical science : AMS · 2011Article
Corrections and comments
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Authors and funding
1 author at 1 institution in 1 country.
Funding
Abstract
Spinal muscular atrophy is an autosomal recessive motor neuron disease that is the leading inherited cause of infant and early childhood mortality. Spinal muscular atrophy is caused by mutation of the telomeric copy of the survival motor neuron gene (SMN1), but all patients retain a centromeric copy of the gene, SMN2. SMN2 produces reduced amounts of full-length SMN mRNA, and spinal muscular atrophy likely results from insufficient levels of SMN protein in motor neurons. The SMN protein plays a well-established role in assembly of the spliceosome and may also mediate mRNA trafficking in the axon and nerve terminus of neurons. In patients, spinal muscular atrophy disease severity correlates inversely with increased SMN2 gene copy number and, in transgenic mice lacking endogenous SMN, increasing SMN2 gene copy number from two to eight prevents the SMA disease phenotype. These observations suggest that increasing SMN expression levels may be beneficial to SMA patients. Currently pursued therapeutic strategies for SMA include induction of SMN2 gene expression, modulation of splicing of SMN2-derived transcripts, stabilization of SMN protein, neuroprotection of SMN deficit neurons, and SMN1 gene replacement. Early clinical trials of candidate therapeutics are now ongoing in SMA patients. Clinical trials in this disease present a unique set of challenges, including the development of meaningful outcome measures and disease biomarkers.
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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.