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Aronin, Neil

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

Property	Value
overview	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 My 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
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: Generation of fusion proteins, raising antisera Preparation of organelles Immunocytochemistry, light microscopy and electron microscopy Immunoprecipitation methods PCR Protein separation techniques, gel electrophoresis, immunoblotting Autoradiography

Property

Value

overview

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

My 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

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:

Generation of fusion proteins, raising antisera
Preparation of organelles
Immunocytochemistry, light microscopy and electron microscopy
Immunoprecipitation methods
PCR
Protein separation techniques, gel electrophoresis, immunoblotting
Autoradiography

One or more keywords matched the following items that are connected to Aronin, Neil

Item Type	Name
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	Alzheimer Disease
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.
Academic Article	Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation.
Academic Article	A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system.
Academic Article	mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs in vivo.
Academic Article	mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo.
Academic Article	Enhancing siRNA efficacy in vivo with extended nucleic acid backbones.
Academic Article	Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation.
Academic Article	Detection of HTTex1p by western blot and immunostaining of HD human and mouse brain using neo-epitope antibody P90 highlights impact of CAG repeat expansion on its size, solubility, and response to MSH3 silencing.

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