Antisense oligonucleotides and their applications in rare neurological diseases
McDowall S, Aung-Htut M, Wilton S, Li D. Antisense oligonucleotides and their applications in rare neurological diseases. Frontiers In Neuroscience 2024, 18: 1414658. PMID: 39376536, PMCID: PMC11456401, DOI: 10.3389/fnins.2024.1414658.Peer-Reviewed Original ResearchAntisense oligonucleotidesRare diseaseImpaired quality of lifeDuchenne muscular dystrophyRare neurological diseasesNeurological diseasesClinical trial designSpinal muscular atrophyRare conditionApplication of antisense oligonucleotidesHigh treatment costsTherapeutic antisense oligonucleotidesClinical trialsAnimal modelsMuscular dystrophyEffective treatmentQuality of lifeImpaired qualityMuscular atrophyAntisense oligomersTrial designTherapeutic validityTarget gene expressionDiseaseTreatment costs
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