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Synergistic Effect of an Antisense Oligonucleotide and Small Molecule on Splicing Correction of the Spinal Muscular Atrophy Gene

Neuroscience Insights(2024)10.1177/26331055241233596Source: DataRank Database

Synergistic Effect of an Antisense Oligonucleotide and Small Molecule on Splicing Correction of the Spinal Muscular Atrophy Gene is a research paper published in Neuroscience Insights (2024). On theSindex it has a DataRank of 0. It has been cited 8 times.

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Open Access8 citations

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datarank_citation_only_1hop_v6· scope data_onlyMethodology

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Enrichment:Funding (2 grants)OA: goldTopics (40)IDs (PubMed, OpenAlex)SDGs (2)

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Authors (2)

Ravindra N Singh ORCID,Eric W. Ottesen

Live enrichment

PubMed:38379891 OpenAlex:https://openalex.org/W4391935046

National Institute of Neurological Disorders and Stroke

Grant: R01 NS055925

National Institutes of Health

Grant: 5R01NS055925-11

Characterization of a complex regulatory element of Spinal Muscular Atrophy genes

goldLicense: cc-by-nc

journalhttps://journals.sagepub.com/doi/pdf/10.1177/26331055241233596

publisherhttps://journals.sagepub.com/doi/pdf/10.1177/26331055241233596

publisherhttps://journals.sagepub.com/doi/full-xml/10.1177/26331055241233596

journalhttps://doi.org/10.1177/26331055241233596

repositoryhttps://pubmed.ncbi.nlm.nih.gov/38379891

Fields of Study

Neurogenetic and Muscular Disorders ResearchRNA modifications and cancerRNA Research and Splicing0301 basic medicine0303 health sciences03 medical and health sciences

Keywords

Spinal muscular atrophyExonSMA*Exon skippingRNA splicingAlternative splicingTranscriptomeOligonucleotideGeneGene expressionBiologyRNACombination therapyAtrophyMedicinePharmacologyGeneticsComputer scienceDiagnostic and Therapeutic Techniques and Equipment570Risdiplam610Survival motor neuron (SMN)Neurosciences. Biological psychiatry. NeuropsychiatryAnti-N1DegreeDisciplines::Medicine and Health Sciences::AnalyticalSpinal muscular atrophy (SMA)ASOISS-N1NusinersenCommentaryBranaplamAntisense oligonucleotideRC321-571

Sustainable Development Goals

SDG 3: 3. Good healthSDG 0: Good health and well-being