Neuron · 2024 · 23 citations · 46 references
Life SciencesMolecular BiologyNeuroscienceGene ExpressionMolecular NeurobiologyMolecular NeuroscienceMolecular NeurogeneticsNeurodegenerative DiseasesNeuroprotectionRna InterferenceAntisense Oligonucleotide DesignAntisense Oligonucleotide SilencingAntisense RnasHumanized ModelsHumanized Mouse ModelsOligonucleotide Therapeutics
46
Fast and accurate short read alignment with Burrows–Wheeler transform
Heng Li, Richard Durbin · Bioinformatics · 2009 · 62.8K citations · Full text
Predicting Splicing from Primary Sequence with Deep Learning
Kishore Jaganathan, Sofia Kyriazopoulou Panagiotopoulou, Jeremy F. McRae et al. · Cell · 2019 · 2.9K citations · Full text
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 · 2.3K 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 · Full text
Medicine, Neurology, Neuroscience +17