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One or more keywords matched the following properties of Aronin, Neil
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Background

  • Undergraduate: Duke University, 1966-1970
  • Medical School: University of Pennsylvania, 1970-1974
  • Internship and Residency: Duke University Medical Center, 1974-1977
  • Research Fellowship: Massachusetts General Hospital and Harvard MedicalSchool, 1979-1981

Positions

  • University of Massachusetts Medical School, 1981 - present,
  • Professor of Medicine and Cell Biology,
  • Director of Endocrinology and Metabolism

Board Certifications

  • Internal Medicine, Endocrinology and Metabolism

Huntington's Disease

Photo: Neil AroninMy clinical interests focus on neuroendocrinology (pituitary and hypothalamic diseases), inherited endocrine diseases, and adrenal problems. We are currently conducting research in the treatment of patients with impaired pituitary function. My basic research centers on the pathogensis of neurodegenerative diseases, especially Parkinson's disease and Huntington's disease.

Dr. Aronin's Clinical Site This link goes 
to an external web site

Rotation Projects

Potential Rotation Projects

Project #1: Molecular Mechanisms in the Pathogenesis of Neurodegenerative Disease. The student can select projects emphasizing either molecular mechanisms of huntingtin in trafficking in neurons or receptor stimulated effects on gene expression in neurons. Techniques include:

  1. Generation of fusion proteins, raising antisera
  2. Preparation of organelles
  3. Immunocytochemistry, light microscopy and electron microscopy
  4. Immunoprecipitation methods
  5. PCR
  6. Protein separation techniques, gel electrophoresis, immunoblotting
  7. Autoradiography
One or more keywords matched the following items that are connected to Aronin, Neil
Item TypeName
Academic Article Unexpected c-fos gene expression in the suprachiasmatic nucleus of mice entrained to a skeleton photoperiod.
Academic Article Expression of normal and mutant huntingtin in the developing brain.
Academic Article In vitro stimulation of c-Fos protein expression in the suprachiasmatic nucleus of hypothalamic slices.
Academic Article Fast transport and retrograde movement of huntingtin and HAP 1 in axons.
Academic Article Are neuronal intranuclear inclusions the common neuropathology of triplet-repeat disorders with polyglutamine-repeat expansions?
Academic Article Wild-type and mutant huntingtins function in vesicle trafficking in the secretory and endocytic pathways.
Academic Article Axonal transport of N-terminal huntingtin suggests early pathology of corticostriatal projections in Huntington disease.
Academic Article Forskolin and dopamine D1 receptor activation increase huntingtin's association with endosomes in immortalized neuronal cells of striatal origin.
Academic Article Are there multiple pathways in the pathogenesis of Huntington's disease?
Academic Article c-Fos expression in the rat intergeniculate leaflet: photic regulation, co-localization with Fos-B, and cellular identification.
Academic Article Differential regulation of fos family genes in the ventrolateral and dorsomedial subdivisions of the rat suprachiasmatic nucleus.
Academic Article Huntingtin expression stimulates endosomal-lysosomal activity, endosome tubulation, and autophagy.
Academic Article A kindred with a variant of multiple endocrine neoplasia type 1 demonstrating frequent expression of pituitary tumors but not linked to the multiple endocrine neoplasia type 1 locus at chromosome region 11q13.
Academic Article Early and progressive accumulation of reactive microglia in the Huntington disease brain.
Academic Article Dde-I restriction endonuclease fragmentation: a novel method of generating cDNA probes for in situ hybridization in brain.
Academic Article Downregulation of histone H4 gene transcription during postnatal development in transgenic mice and at the onset of differentiation in transgenically derived calvarial osteoblast cultures.
Academic Article Caspase 3-cleaved N-terminal fragments of wild-type and mutant huntingtin are present in normal and Huntington's disease brains, associate with membranes, and undergo calpain-dependent proteolysis.
Academic Article Changes in cortical and striatal neurons predict behavioral and electrophysiological abnormalities in a transgenic murine model of Huntington's disease.
Academic Article Aggregation of huntingtin in neuronal intranuclear inclusions and dystrophic neurites in brain.
Academic Article Huntingtin localization in brains of normal and Huntington's disease patients.
Academic Article Huntingtin is present in the nucleus, interacts with the transcriptional corepressor C-terminal binding protein, and represses transcription.
Academic Article Maintenance of susceptibility to neurodegeneration following intrastriatal injections of quinolinic acid in a new transgenic mouse model of Huntington's disease.
Academic Article Transient myocardial perfusion abnormalities in diabetic patients: a prospective study using thallium exercise tolerance testing.
Academic Article Analysis of Huntingtin-associated protein 1 in mouse brain and immortalized striatal neurons.
Academic Article Mutant huntingtin expression in clonal striatal cells: dissociation of inclusion formation and neuronal survival by caspase inhibition.
Academic Article Huntingtin is degraded to small fragments by calpain after ischemic injury.
Academic Article Asymmetry in the assembly of the RNAi enzyme complex.
Academic Article Striatal potassium channel dysfunction in Huntington's disease transgenic mice.
Academic Article The subcellular localization of the G-protein Gi alpha in the basal ganglia reveals its potential role in both signal transduction and vesicle trafficking.
Academic Article Photoinducible and rhythmic ICER-CREM immunoreactivity in the rat suprachiasmatic nucleus.
Academic Article Novel pheochromocytoma susceptibility loci identified by integrative genomics.
Academic Article Lysosomal proteases are involved in generation of N-terminal huntingtin fragments.
Academic Article Target selectivity in mRNA silencing.
Academic Article Coordinate occupancy of AP-1 sites in the vitamin D-responsive and CCAAT box elements by Fos-Jun in the osteocalcin gene: model for phenotype suppression of transcription.
Academic Article Designing siRNA that distinguish between genes that differ by a single nucleotide.
Academic Article Pro-caspase-8 is predominantly localized in mitochondria and released into cytoplasm upon apoptotic stimulation.
Academic Article Fluorescent and biotin probes for dopamine receptors: D1 and D2 receptor affinity and selectivity.
Academic Article Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits.
Academic Article Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients.
Academic Article Expanded CAG repeats in the crosshairs.
Academic Article A Golgi study of the monkey paraventricular nucleus: neuronal types, afferent and efferent fibers.
Academic Article Polyglutamine expansion in huntingtin alters its interaction with phospholipids.
Academic Article Mutant huntingtin and glycogen synthase kinase 3-beta accumulate in neuronal lipid rafts of a presymptomatic knock-in mouse model of Huntington's disease.
Academic Article Mutant huntingtin impairs vesicle formation from recycling endosomes by interfering with Rab11 activity.
Academic Article Germline mutations in TMEM127 confer susceptibility to pheochromocytoma.
Academic Article Aberrant Rab11-dependent trafficking of the neuronal glutamate transporter EAAC1 causes oxidative stress and cell death in Huntington's disease.
Academic Article Oligonucleotide therapeutic approaches for Huntington disease.
Academic Article Argonaute protein identity and pairing geometry determine cooperativity in mammalian RNA silencing.
Academic Article Frameless multimodal image guidance of localized convection-enhanced delivery of therapeutics in the brain.
Academic Article Native mutant huntingtin in human brain: evidence for prevalence of full-length monomer.
Academic Article Does capsaicin relieve the pain of diabetic neuropathy?
Academic Article Striatal neurochemical changes in transgenic models of Huntington's disease.
Academic Article Synaptic interactions between GABAergic neurons and trigeminothalamic cells in the rat trigeminal nucleus caudalis.
Academic Article Increased huntingtin protein length reduces the number of polyglutamine-induced gene expression changes in mouse models of Huntington's disease.
Academic Article Immunoreactive calcium-binding protein (calbindin-D28k) in interneurons and trigeminothalamic neurons of the rat nucleus caudalis localized with peroxidase and immunogold methods.
Academic Article Hunting down huntingtin.
Academic Article Ultrastructural localization of immunoreactive calbindin-D28k in the rat and monkey basal ganglia, including subcellular distribution with colloidal gold labeling.
Academic Article siRNAs knock down hepatitis.
Academic Article Glutamic acid decarboxylase and enkephalin immunoreactive axon terminals in the rat neostriatum synapse with striatonigral neurons.
Academic Article N-methyl-D-aspartate receptor activation in the neostriatum increases c-fos and fos-related antigens selectively in medium-sized neurons.
Academic Article Autophagy regulates the processing of amino terminal huntingtin fragments.
Academic Article Light and electron microscopic localization of immunoreactive Leu-enkephalin in the monkey basal ganglia.
Academic Article Immunoreactive leu-enkephalin in the monkey hypothalamus including observations on its ultrastructural localization in the paraventricular nucleus.
Academic Article Immunoreactive substance P in the substantia nigra of the monkey: light and electron microscopic localization.
Academic Article Substance P and neurotensin.
Academic Article Regulation by thyroid hormone of the concentration of substance P in the rat anterior pituitary.
Academic Article The effects of gonadal steroids on the content of substance P in the rat anterior pituitary.
Academic Article Ultrastructural localization and biochemical features of immunoreactive LEU-enkephalin in monkey dorsal horn.
Academic Article Localization of immunoreactive enkephalins in GABA synthesizing neurons of the rat neostriatum.
Academic Article Huntingtin bodies sequester vesicle-associated proteins by a polyproline-dependent interaction.
Academic Article The stability and metabolism of intravenously administered neurotensin in the rat.
Academic Article Monoamines in the brain cerebrospinal fluid of facial pain patients.
Academic Article Functional role for Sp1 in the transcriptional amplification of a cell cycle regulated histone H4 gene.
Academic Article Temporal regulation of light-induced Fos and Fos-like protein expression in the ventrolateral subdivision of the rat suprachiasmatic nucleus.
Academic Article Colocalization of D1 and D2 dopamine receptor mRNAs in striatal neurons.
Academic Article Immunohistochemical localization of the D1 dopamine receptor in rat brain reveals its axonal transport, pre- and postsynaptic localization, and prevalence in the basal ganglia, limbic system, and thalamic reticular nucleus.
Academic Article The myopathy of Cushing's syndrome.
Academic Article Huntingtin associates with acidic phospholipids at the plasma membrane.
Academic Article Light regulates expression of a Fos-related protein in rat suprachiasmatic nuclei.
Academic Article Diabetes mellitus and silent myocardial ischemia.
Academic Article A function of huntingtin in guanine nucleotide exchange on Rab11.
Academic Article Linking SNPs to CAG repeat length in Huntington's disease patients.
Academic Article The differential effects of thyroid and gonadal hormones on substance P content in the anterior pituitary of the prepubertal rat.
Academic Article Substance P and neurotensin: their roles in the regulation of anterior pituitary function.
Academic Article Diminished flare response in neuropathic diabetic patients. Comparison of effects of substance P, histamine, and capsaicin.
Academic Article Hypothyroidism increases substance P concentrations in the heterotopic anterior pituitary.
Academic Article Disruption of Rab11 activity in a knock-in mouse model of Huntington's disease.
Academic Article Huntingtin cleavage product A forms in neurons and is reduced by gamma-secretase inhibitors.
Academic Article Immunohistochemical evidence for neural mediation of VIP activity in the dogfish rectal gland.
Academic Article Frequency of painless myocardial ischemia during exercise tolerance testing in patients with and without diabetes mellitus.
Academic Article Ultrastructural features of immunoreactive somatostatin neurons in the rat caudate nucleus.
Academic Article Quantitative electron microscopic study of immunoreactive somatostatin axons in the rat neostriatum.
Academic Article Light microscopic and ultrastructural localization of immunoreactive substance P in the dorsal horn of monkey spinal cord.
Academic Article Somatostatin is increased in the basal ganglia in Huntington disease.
Academic Article The distribution of substance P-containing neurons in the cat Edinger-Westphal nucleus: relationship to efferent projection systems.
Academic Article Sustained remission of Nelson's syndrome after stopping cyproheptadine treatment.
Academic Article Impaired clearance of beta-lipotropin in uremia.
Academic Article Comparative metabolic clearance rates of beta-endorphin and beta-lipotropin in humans.
Academic Article Ultrastructural localization of immunoreactive neurotensin in the monkey superficial dorsal horn.
Academic Article Environmental iodine intake and thyroid dysfunction during chronic amiodarone therapy.
Academic Article Prognostic differences in subgroups of patients with electrographic evidence of subendocardial or transmural myocardial infarction. The favorable outlook for patients with an initially normal QRS complex.
Academic Article CAG expansion affects the expression of mutant Huntingtin in the Huntington's disease brain.
Academic Article Circadian regulation of Fos B is different from c-Fos in the rat suprachiasmatic nucleus.
Academic Article Compositional changes of AP-1 DNA-binding proteins are regulated by light in a mammalian circadian clock.
Concept Tandem Mass Spectrometry
Concept Drug Stability
Concept Disease Models, Animal
Concept Sequence Analysis, DNA
Concept Central Nervous System
Concept Subcellular Fractions
Concept Synaptosomes
Concept Genes, Regulator
Concept RNA, Small Interfering
Concept Mass Spectrometry
Concept Genetic Association Studies
Concept Software
Concept Photic Stimulation
Concept Motor Neuron Disease
Concept Oncogenes
Concept Oxidative Stress
Concept Rats, Inbred Strains
Concept HSC70 Heat-Shock Proteins
Concept Deoxyribonucleases, Type II Site-Specific
Concept Surgery, Computer-Assisted
Concept Polymorphism, Single Nucleotide
Concept Disease Progression
Concept Sensitivity and Specificity
Concept Stereotaxic Techniques
Concept Cell Survival
Concept Proto-Oncogenes
Concept Genes, Dominant
Concept Disease Susceptibility
Concept Neurodegenerative Diseases
Concept Genes
Concept RNA-Induced Silencing Complex
Concept Synaptic Transmission
Concept Amino Acid Sequence
Concept Drug Delivery Systems
Concept Stress, Physiological
Concept Sexual Maturation
Concept Suppression, Genetic
Concept Cohort Studies
Concept Coronary Artery Disease
Concept Genes, fos
Concept Receptors, Serotonin
Concept Skin Tests
Concept Injections, Spinal
Concept Genetic Predisposition to Disease
Concept Synaptic Vesicles
Concept Remission, Spontaneous
Concept Cell-Free System
Concept Sciatic Nerve
Concept Static Electricity
Concept Molecular Structure
Concept Immune Sera
Concept Transgenes
Concept Muscular Diseases
Concept Regulatory Sequences, Nucleic Acid
Concept Retrospective Studies
Concept Repetitive Sequences, Nucleic Acid
Concept DNA Repeat Expansion
Concept RNA Stability
Concept Species Specificity
Concept Parasympathetic Nervous System
Concept Huntington Disease
Concept Sheep, Domestic
Concept Swine, Miniature
Concept Serotonin Plasma Membrane Transport Proteins
Concept Signal Transduction
Concept Suprachiasmatic Nucleus
Concept Nucleic Acid Conformation
Concept Ganglia, Spinal
Concept Spinal Cord
Concept Somatostatin
Concept Superoxide Dismutase
Concept High-Throughput Screening Assays
Concept Calorimetry, Differential Scanning
Concept Reactive Oxygen Species
Concept Protein Structure, Tertiary
Concept Adaptor Proteins, Signal Transducing
Concept Solubility
Concept Substance P
Concept Genes, Reporter
Concept Glycogen Synthase Kinase 3
Concept Synapses
Concept Electric Stimulation
Concept Sex Factors
Concept Trinucleotide Repeat Expansion
Concept Sharks
Concept Sheep
Concept Thyroid Diseases
Concept Coronary Disease
Concept Genetic Carrier Screening
Concept Genome, Human
Concept Binding Sites
Concept Mass Screening
Concept Salt Gland
Concept Spheroids, Cellular
Concept Quantitative Trait Loci
Concept Digestive System
Concept Mice, Inbred Strains
Concept Cushing Syndrome
Concept Antibody Specificity
Concept Prospective Studies
Concept Sequence Homology, Amino Acid
Concept Skull
Concept Staining and Labeling
Concept Sequence Homology, Nucleic Acid
Concept In Situ Hybridization
Concept Substrate Specificity
Concept Sp1 Transcription Factor
Concept Embryonic Stem Cells
Concept Oligonucleotide Array Sequence Analysis
Concept Rats, Sprague-Dawley
Concept Limbic System
Concept Swine
Concept Corpus Striatum
Concept Serine
Concept In Situ Hybridization, Fluorescence
Concept Substantia Nigra
Concept Gene Silencing
Concept Base Sequence
Concept Alleles
Concept Sodium Oxybate
Concept Follow-Up Studies
Concept Heart Diseases
Concept Genome, Viral
Concept Nelson Syndrome
Concept Molecular Sequence Data
Concept Genes, Immediate-Early
Concept RNA, Double-Stranded
Concept Synapsins
Academic Article A synthetic luciferin improves bioluminescence imaging in live mice.
Academic Article Striatal synaptosomes from Hdh140Q/140Q knock-in mice have altered protein levels, novel sites of methionine oxidation, and excess glutamate release after stimulation.
Academic Article HTT-lowering reverses Huntington's disease immune dysfunction caused by NF?B pathway dysregulation.
Academic Article Increased Steady-State Mutant Huntingtin mRNA in Huntington's Disease Brain.
Academic Article Assessment of chloroquine treatment for modulating autophagy flux in brain of WT and HD mice.
Academic Article Finding the striatum in sheep: use of a multi-modal guided approach for convection enhanced delivery.
Academic Article CRISPRseek: a bioconductor package to identify target-specific guide RNAs for CRISPR-Cas9 genome-editing systems.
Academic Article Huntingtin-lowering strategies in Huntington's disease: antisense oligonucleotides, small RNAs, and gene editing.
Academic Article Mass Spectrometry Analysis of Wild-Type and Knock-in Q140/Q140 Huntington's Disease Mouse Brains Reveals Changes in Glycerophospholipids Including Alterations in Phosphatidic Acid and Lyso-Phosphatidic Acid.
Academic Article Safety of Striatal Infusion of siRNA in a Transgenic Huntington's Disease Mouse Model.
Academic Article Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain.
Academic Article Excitatory transmission onto AgRP neurons is regulated by cJun NH2-terminal kinase 3 in response to metabolic stress.
Academic Article Does the Mutant CAG Expansion in Huntingtin mRNA Interfere with Exonucleolytic Cleavage of its First Exon?
Academic Article A High-Throughput Method for Direct Detection of Therapeutic Oligonucleotide-Induced Gene Silencing In Vivo.
Academic Article Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector.
Academic Article Recurrent Mutations of Chromatin-Remodeling Genes and Kinase Receptors in Pheochromocytomas and Paragangliomas.
Academic Article Effects of Exogenous NUB1 Expression in the Striatum of HDQ175/Q7 Mice.
Academic Article Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain.
Academic Article Autophagy Activation by Transcription Factor EB (TFEB) in Striatum of HDQ175/Q7 Mice.
Academic Article Cellular Analysis of Silencing the?Huntington's Disease Gene Using AAV9?Mediated Delivery of Artificial Micro?RNA into the Striatum of?Q140/Q140?Mice.
Academic Article Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing.
Academic Article High-resolution proteomic and lipidomic analysis of exosomes and microvesicles from different cell sources.
Academic Article Allele-Selective Suppression of Mutant Huntingtin in Primary Human Blood Cells.
Academic Article 5?-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo.
Academic Article Synthesis and Evaluation of Parenchymal Retention and Efficacy of a Metabolically Stable O-Phosphocholine-N-docosahexaenoyl-l-serine siRNA Conjugate in Mouse Brain.
Academic Article Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin.
Academic Article Nuclear Localization of Huntingtin mRNA Is Specific to Cells of Neuronal Origin.
Academic Article Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer.
Academic Article Alterations in mRNA 3' UTR Isoform Abundance Accompany Gene Expression Changes in Human Huntington's Disease Brains.
Academic Article Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo.
Academic Article Hydrophobicity of Lipid-Conjugated siRNAs Predicts Productive Loading to Small Extracellular Vesicles.
Academic Article A Fresh Look at Huntingtin mRNA Processing in Huntington's Disease.
Academic Article Optimized Cholesterol-siRNA Chemistry Improves Productive Loading onto Extracellular Vesicles.
Academic Article Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease.
Academic Article Chitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brain.
Academic Article Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier.
Academic Article Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration.
Academic Article A High-throughput Assay for mRNA Silencing in Primary Cortical Neurons in vitro with Oligonucleotide Therapeutics.
Academic Article IMPROVING AFTER-HOURS CALLS TO ENDOCRINOLOGY FELLOWS.
Academic Article Loading of Extracellular Vesicles with Hydrophobically Modified siRNAs.
Academic Article Exosomes Produced from 3D Cultures of MSCs by Tangential Flow Filtration Show Higher Yield and Improved Activity.
Academic Article Loading of Extracellular Vesicles with Chemically Stabilized Hydrophobic siRNAs for the Treatment of Disease in the Central Nervous System.
Academic Article Rac1 Activity Is Modulated by Huntingtin and Dysregulated in Models of Huntington's Disease.
Academic Article Huntingtin associates with the actin cytoskeleton and a-actinin isoforms to influence stimulus dependent morphology changes.
Academic Article Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition.
Academic Article A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.
Academic Article Ly6a Differential Expression in Blood-Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B.
Academic Article A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna.
Academic Article Huntington's Disease: Les Jeux Sont Faits?
Academic Article Large Animal Models of Huntington's Disease: What We Have Learned and Where We Need to Go Next.
Academic Article Genotype-Phenotype Features of Germline Variants of the TMEM127 Pheochromocytoma Susceptibility Gene: A 10-Year Update.
Academic Article Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities.
Academic Article Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity.
Academic Article Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep.
Concept Superoxide Dismutase-1
Concept Exome
Concept Serogroup
Concept Infusions, Spinal
Concept S100 Calcium Binding Protein G
Concept CRISPR-Cas Systems
Academic Article Perspective: Tominersen Testing Finds a Way Forward.
Academic Article Mutant huntingtin messenger RNA forms neuronal nuclear clusters in rodent and human brains.
Academic Article Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models.
Academic Article Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's disease.
Academic Article Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo.
Academic Article Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo.
Academic Article Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's disease.
Academic Article Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model.
Academic Article A programmable dual-targeting di-valent siRNA scaffold supports potent multi-gene modulation in the central nervous system.
Academic Article Nacc1 Mutation in Mice Models Rare Neurodevelopmental Disorder with Underlying Synaptic Dysfunction.
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