ArticleMolecular therapy. Nucleic acids2013
Lipid Nanoparticle Delivery of siRNA to Silence Neuronal Gene Expression in the Brain.
Article in Molecular therapy. Nucleic acids, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 78 papers.
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Who cites it
78 citing papers in PubMed, 161 citations in OpenAlex.
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- Antisense oligonucleotide therapy for patients with Friedreich's ataxia carrying the c.165+5G>C splicing mutation.Molecular therapy. Nucleic acids · 2025Article
- Knocking on Cells' Door: Strategic Approaches for miRNA and siRNA in Anticancer Therapy.International journal of molecular sciences · 2025Review
- mRNA lipid nanoparticle formulation, characterization and evaluation.Nature protocols · 2025Review
- Xist condensates: perspectives for therapeutic intervention.Genome biology · 2025Review
- Therapeutic Delivery of circDYM by Perillyl Alcohol Nanoemulsion Alleviates LPS-Induced Depressive-Like Behaviors.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
- Use of polyadenosine tail mimetics to enhance mRNA expression from genes associated with haploinsufficiency disorders.Molecular therapy. Nucleic acids · 2025Article
- Comprehensive analysis of lipid nanoparticle formulation and preparation for RNA delivery.International journal of pharmaceutics: X · 2024Review
- Insulin-inspired hippocampal neuron-targeting technology for protein drug delivery.Proceedings of the National Academy of Sciences of the United States of America · 2024Article
- Unleashing the potential of mRNA therapeutics for inherited neurological diseases.Brain : a journal of neurology · 2024Review
- The 60-year evolution of lipid nanoparticles for nucleic acid delivery.Nature reviews. Drug discovery · 2024Review
- Multifunctional Nanocarriers for Alzheimer's Disease: Befriending the Barriers.Molecular neurobiology · 2024Review
- Ionic liquid-coated lipid nanoparticles increase siRNA uptake into CNS targets.Nanoscale advances · 2024Article
- Restoring the Function of Thalamocortical Circuit Through Correcting Thalamic Kv3.2 Channelopathy Normalizes Fear Extinction Impairments in a PTSD Mouse Model.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2024Article
- Lipid-based nanoparticles for drug delivery in Parkinson's disease.Translational neuroscience · 2024Review
18 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
9 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Manipulation of gene expression in the brain is fundamental for understanding the function of proteins involved in neuronal processes. In this article, we show a method for using small interfering RNA (siRNA) in lipid nanoparticles (LNPs) to efficiently silence neuronal gene expression in cell culture and in the brain in vivo through intracranial injection. We show that neurons accumulate these LNPs in an apolipoprotein E-dependent fashion, resulting in very efficient uptake in cell culture (100%) with little apparent toxicity. In vivo, intracortical or intracerebroventricular (ICV) siRNA-LNP injections resulted in knockdown of target genes either in discrete regions around the injection site or in more widespread areas following ICV injections with no apparent toxicity or immune reactions from the LNPs. Effective targeted knockdown was demonstrated by showing that intracortical delivery of siRNA against GRIN1 (encoding GluN1 subunit of the NMDA receptor (NMDAR)) selectively reduced synaptic NMDAR currents in vivo as compared with synaptic AMPA receptor currents. Therefore, LNP delivery of siRNA rapidly manipulates expression of proteins involved in neuronal processes in vivo, possibly enabling the development of gene therapies for neurological disorders.Molecular Therapy-Nucleic Acids (2013) 2, e136; doi:10.1038/mtna.2013.65; published online 3 December 2013.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.