Antisense oligonucleotides: the next frontier for treatment of neurological disorders

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Pharmacology of a Central Nervous System Delivered 2′-O-Methoxyethyl–Modified Survival of Motor Neuron Splicing Oligonucleotide in Mice and Nonhuman Primates

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C9orf72 hypermethylation protects against repeat expansion-associated pathology in ALS/FTD

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Antisense oligonucleotides: treating neurodegeneration at the level of RNA

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The evolution of commercial drug delivery technologies

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Smart nanoparticles for cancer therapy

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The blood-brain barrier: Physiology and strategies for drug delivery

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Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for <i>SOD1</i> ALS

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A Quarter Century of APOE and Alzheimer’s Disease: Progress to Date and the Path Forward

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Therapies for rare diseases: therapeutic modalities, progress and challenges ahead

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Convective influx/glymphatic system: tracers injected into the CSF enter and leave the brain along separate periarterial basement membrane pathways

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Structure-Based Discovery of SD-36 as a Potent, Selective, and Efficacious PROTAC Degrader of STAT3 Protein

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Neurotransmitter-derived lipidoids (NT-lipidoids) for enhanced brain delivery through intravenous injection

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Efficient Delivery of Antisense Oligonucleotides Using Bioreducible Lipid Nanoparticles In Vitro and In Vivo

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Therapeutic reversal of Huntington’s disease by <i>in vivo</i> self-assembled siRNAs

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Altered cell and RNA isoform diversity in aging Down syndrome brains

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Antisense oligonucleotide therapy for KCNT1 encephalopathy

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Recent advances in gene therapy for neurodevelopmental disorders with epilepsy

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Advances in treatment of Dyslipidemia

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Retinal disease in ciliopathies: Recent advances with a focus on stem cell-based therapies

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Clinical development of passive tau-based immunotherapeutics for treating primary and secondary tauopathies

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Designing poly-agonists for treatment of metabolic diseases: challenges and opportunities

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Reduction of nemo-like kinase increases lysosome biogenesis and ameliorates TDP-43–related neurodegeneration

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