| http://purl.uniprot.org/citations/29552580 | http://www.w3.org/1999/02/22-rdf-syntax-ns#type | http://purl.uniprot.org/core/Journal_Citation |
| http://purl.uniprot.org/citations/29552580 | http://www.w3.org/2000/01/rdf-schema#comment | "Spinal muscular atrophy (SMA) is a devastating childhood motor neuron disease. SMA is caused by mutations in the survival motor neuron gene (SMN1), leading to reduced levels of SMN protein in the CNS. The actin-binding protein plastin 3 (PLS3) has been reported as a modifier for SMA, making it a potential therapeutic target. Here, we show reduced levels of PLS3 protein in the brain and spinal cord of a mouse model of SMA. Our study also revealed that lentiviral-mediated PLS3 expression restored axonal length in cultured Smn-deficient motor neurons. Delivery of adeno-associated virus serotype 9 (AAV9) harboring Pls3 cDNA via cisterna magna in SMNΔ7 mice, a widely used animal model of SMA, led to high neuronal transduction efficiency. PLS3 treatment allowed a small but significant increase of lifespan by 42%. Although there was no improvement of phenotype, this study has demonstrated the potential use of Pls3 as a target for gene therapy, possibly in combination with other disease modifiers."xsd:string |
| http://purl.uniprot.org/citations/29552580 | http://purl.org/dc/terms/identifier | "doi:10.1016/j.omtm.2018.01.007"xsd:string |
| http://purl.uniprot.org/citations/29552580 | http://purl.uniprot.org/core/author | "Ning K."xsd:string |
| http://purl.uniprot.org/citations/29552580 | http://purl.uniprot.org/core/author | "Lewis K.E."xsd:string |
| http://purl.uniprot.org/citations/29552580 | http://purl.uniprot.org/core/author | "Azzouz M."xsd:string |
| http://purl.uniprot.org/citations/29552580 | http://purl.uniprot.org/core/author | "Iremonger K."xsd:string |
| http://purl.uniprot.org/citations/29552580 | http://purl.uniprot.org/core/author | "Karyka E."xsd:string |
| http://purl.uniprot.org/citations/29552580 | http://purl.uniprot.org/core/author | "Alrafiah A."xsd:string |
| http://purl.uniprot.org/citations/29552580 | http://purl.uniprot.org/core/author | "Coldicott I."xsd:string |
| http://purl.uniprot.org/citations/29552580 | http://purl.uniprot.org/core/date | "2018"xsd:gYear |
| http://purl.uniprot.org/citations/29552580 | http://purl.uniprot.org/core/name | "Mol Ther Methods Clin Dev"xsd:string |
| http://purl.uniprot.org/citations/29552580 | http://purl.uniprot.org/core/pages | "81-89"xsd:string |
| http://purl.uniprot.org/citations/29552580 | http://purl.uniprot.org/core/title | "Plastin 3 Promotes Motor Neuron Axonal Growth and Extends Survival in a Mouse Model of Spinal Muscular Atrophy."xsd:string |
| http://purl.uniprot.org/citations/29552580 | http://purl.uniprot.org/core/volume | "9"xsd:string |
| http://purl.uniprot.org/citations/29552580 | http://www.w3.org/2004/02/skos/core#exactMatch | http://purl.uniprot.org/pubmed/29552580 |
| http://purl.uniprot.org/citations/29552580 | http://xmlns.com/foaf/0.1/primaryTopicOf | https://pubmed.ncbi.nlm.nih.gov/29552580 |
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| http://purl.uniprot.org/uniprot/#_B8JJX7-mappedCitation-29552580 | http://www.w3.org/1999/02/22-rdf-syntax-ns#object | http://purl.uniprot.org/citations/29552580 |
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| http://purl.uniprot.org/uniprot/#_G3UYV0-mappedCitation-29552580 | http://www.w3.org/1999/02/22-rdf-syntax-ns#object | http://purl.uniprot.org/citations/29552580 |