Precision Medicine through Antisense Oligonucleotide-Mediated Exon Skipping
Li D, Mastaglia F, Fletcher S, Wilton S. Precision Medicine through Antisense Oligonucleotide-Mediated Exon Skipping. Trends In Pharmacological Sciences 2018, 39: 982-994. PMID: 30282590, DOI: 10.1016/j.tips.2018.09.001.Peer-Reviewed Original ResearchConceptsSpinal muscular atrophyAntisense oligonucleotidesAntisense oligonucleotide-mediated exon skippingDuchenne MDTreat Duchenne muscular dystrophyExon skippingTherapeutic potential of antisense oligonucleotidesTargeted exon skippingPotential of antisense oligonucleotidesDuchenne muscular dystrophyPrecision medicineInherited rare diseaseClinical benefitRare diseaseMuscular dystrophyClinical implementationTherapeutic potentialMuscular atrophyAffected individualsAlternative splicingLethal mutationsDuchenneBecker casesExonMutations
This site is protected by hCaptcha and its Privacy Policy and Terms of Service apply