Nature Biotechnology · 2021 · 136 citations · 46 references
Knockout MouseChromatinNeurodegenerative DiseasesMolecular NeuroscienceNeurogenomicsBlood–brain BarrierMedicineNatural SciencesNucleic Acid BiochemistryOligonucleotideMolecular BiologyCholesterol-functionalized Dna/rna HeteroduplexesMolecular NeurobiologyRodent CnsEpigenetic RegulationCell BiologyEpigeneticsNeurogenetics
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Mutation of the mouse klotho gene leads to a syndrome resembling ageing.
PubMed · 1997 · 66.5K citations
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Richard S. Finkel, Eugenio Mercuri, Basil T. Darras et al. · New England Journal of Medicine · 2017 · 2.2K citations · Full text
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
Eugenio Mercuri, Basil T. Darras, Claudia A. Chiriboga et al. · New England Journal of Medicine · 2018 · 1.4K citations