Molecular Therapy · 2018 · 80 citations · 42 references
PharmacyMolecular BiologyNeuroscienceDrug DeliveryGene DeliveryMolecular NeuroscienceBlood-brain Barrier BiologyBlood-brain Barrier DeliveryBlood-brain Barrier PenetrationIn Vivo Gene DeliveryNucleic Acid DeliverySmall Interfering Rna DeliveryBlood-brain Barrier PermeabilityBlood-brain Barrier TraffickingBlood-brain Barrier Transport StudiesGene Delivery Across The Blood-brain BarrierGene Delivery To The BrainNucleic Acid Delivery To The Central Nervous SystemTargeted Delivery Across The Blood-brain BarrierTargeted Gene Silencing
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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
Mfsd2a is critical for the formation and function of the blood–brain barrier
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