Widespread suppression of huntingtin with convection-enhanced delivery of siRNA.

No abstract is available for this paper.

• Publication year

  2012

• Venue

  Experimental Neurology

• Publication date

  Unknown publication date

• Fields of study

  Biology, Medicine, Engineering

• Identifiers
  DOI 10.1016/j.expneurol.2011.11.020PMID 22119622
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

• Huntington's disease: from molecular pathogenesis to clinical treatment

  2011cited by this paper
• Image-guided convection-enhanced delivery of muscimol to the primate brain.

  2010cited by this paper
• Targeted therapy for high-grade glioma with the TGF-β2 inhibitor trabedersen: results of a randomized and controlled phase IIb study

  2010influential reference
• Real-time MR imaging of adeno-associated viral vector delivery to the primate brain.

  2009cited by this paper
• Diffusion-weighted versus volumetric imaging of the striatum in early symptomatic Huntington disease

  2009influential reference
• Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice.

  2009influential reference
• Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients.

  2009cited by this paper
• A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference.

  2009cited by this paper
• Sustained effects of nonallele‐specific Huntingtin silencing

  2009influential reference
• Convection-Enhanced Delivery for the Treatment of Pediatric Neurologic Disorders

  2008cited by this paper
• Transvascular delivery of small interfering RNA to the central nervous system

  2007cited by this paper
• Image-guided, direct convective delivery of glucocerebrosidase for neuronopathic Gaucher disease

  2007influential reference
• Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits

  2007cited by this paper
• Huntington's disease.

  2007cited by this paper
• RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model.

  2005cited by this paper
• Neurochemical and behavioral consequences of widespread gene knockdown in the adult mouse brain by using nonviral RNA interference.

  2004cited by this paper
• Intraputamenal infusion of GDNF in aged rhesus monkeys: Distribution and dopaminergic effects

  2003cited by this paper
• Imaging‐guided convection‐enhanced delivery and gene therapy of glioblastoma

  2003cited by this paper
• Chronic Intracerebral Delivery of Trophic Factors via a Programmable Pump as a Treatment for Parkinsonism.

  2001cited by this paper
• Striatal volume loss in HD as measured by MRI and the influence of CAG repeat

  2001influential reference
• Age-related decline in striatal volume in monkeys as measured by magnetic resonance imaging.

  2000influential reference
• Comparison of 14C-sucrose delivery to the brain by intravenous, intraventricular, and convection-enhanced intracerebral infusion.

  1999cited by this paper
• Convection-enhanced delivery of macromolecules in the brain.

  1994cited by this paper
• A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. The Huntington's Disease Collaborative Research Group.

  1993cited by this paper
• Huntington's disease

  1984cited by this paper
• Chemical Pathology of the Nervous System (Proceedings of the Third International Neurochemical Symposium held at Strasbourg, 1958)

  1962cited by this paper

• Convection-enhanced delivery for brain malignancies: Technical parameters, formulation strategies and clinical perspectives.

  2025cites this paper
• TfR1-Binding Peptide Conjugation Facilitates Robust and Specific siRNA Delivery to the Central Nervous System.

  2025cites this paper
• RNA-Targeting CRISPR/CasRx system relieves disease symptoms in Huntington’s disease models

  2025cites this paper
• Advances in gene and cellular therapeutic approaches for Huntington’s disease

  2024cites this paper
• Using gene or cell therapies to treat Huntington's disease.

  2024cites this paper
• Global huntingtin knockout in adult mice leads to fatal neurodegeneration that spares the pancreas

  2024cites this paper
• Nanotherapeutic treatment of the invasive glioblastoma tumor microenvironment.

  2022cites this paper
• Insights into White Matter Defect in Huntington’s Disease

  2022cites this paper
• Antisense Oligonucleotide Therapy: From Design to the Huntington Disease Clinic

  2022cites this paper
• Potential disease-modifying therapies for Huntington's disease: lessons learned and future opportunities.

  2022cites this paper
• Gene Therapy for Huntington’s Disease: The Final Strategy for a Cure?

  2021cites this paper
• The Use of Peptide and Protein Vectors to Cross the Blood-Brain Barrier for the Delivery of Therapeutic Concentration of Biologics

  2020cites this paper
• Therapeutic strategies for Huntington's disease.

  2020cites this paper
• Convection-enhanced delivery of liposomal drugs for effective treatment of glioblastoma multiforme

  2020cites this paper
• Huntingtin Lowering Strategies

  2020cites this paper
• Huntingtin-Lowering Therapies for Huntington Disease: A Review of the Evidence of Potential Benefits and Risks.

  2020cites this paper
• Recent Advances in the Treatment of Huntington’s Disease: Targeting DNA and RNA

  2020cites this paper
• The quest towards understanding the molecular pathogenesis of triplet repeat disorders: Huntingtons Disease and Fragile X-Associated Tremor and Ataxia Syndrome

  2020cites this paper
• The effects of huntingtin-lowering: what do we know so far?

  2019cites this paper
• THERAPEUTIC MICRORNAS TARGETING NUCLEAR HUNTINGTIN FOR A DEVELOPMENT OF A PERSONALIZED GENE THERAPY FOR HUNTINGTON’S DISEASE

  2019cites this paper
• Doubling down on siRNAs in the brain

  2019cites this paper
• Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease

  2019cites this paper
• The Emerging Role of microRNAs in Polyglutamine Diseases

  2019cites this paper
• Nonhuman Primate Models of Huntington's Disease and Their Application in Translational Research.

  2018cites this paper
• The CAG-polyglutamine repeat diseases: a clinical, molecular, genetic, and pathophysiologic nosology.

  2018cites this paper
• How will the field of gene therapy survive its success?

  2018cites this paper
• AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model

  2018cites this paper
• Surface modified cubosomes for drug delivery across the blood-brain barrier

  2018cites this paper
• Practical Considerations of Convection Enhanced Delivery for Novel Therapies Treating Spinal Cord Injury Related Neuropathic Pain

  2018cites this paper
• Gene Therapies for Polyglutamine Diseases.

  2018cites this paper
• Polyglutamine Disorders

  2018cites this paper
• AAV 5-miHTT gene therapy demonstrates broad distribution and strong human mutant huntingtin lowering in a Huntington disease minipig model

  2018cites this paper
• siRNA delivery for treatment of degenerative diseases, new hopes and challenges

  2018cites this paper
• Translation of MicroRNA-Based Huntingtin-Lowering Therapies from Preclinical Studies to the Clinic.

  2018cites this paper
• Designing Polymer Micelles of Controlled Size, Stability, and Functionality for siRNA Delivery

  2017cites this paper
• Therapies targeting DNA and RNA in Huntington's disease.

  2017cites this paper
• AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease

  2017cites this paper
• Gene suppression approaches to neurodegeneration

  2017cites this paper
• Continuous intranigral infusion is not associated with observable behavioral deficits or marked pathology: a preclinical safety study.

  2017cites this paper
• Huntington's Disease and Other Choreas

  2017cites this paper
• From huntingtin gene to Huntington’s disease-altering strategies

  2017cites this paper
• [Hope for Huntington's disease patients: first clinical gene silencing study in progress].

  2017cites this paper
• Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease

  2016cites this paper
• Novel targets for Huntington’s disease: future prospects

  2016cites this paper
• Antisense Oligonucleotide Tool in Therapy for Polyglutamine Neurodegenerative Disorders

  2016cites this paper
• Biodegradable DNA Nanoparticles that Provide Widespread Gene Delivery in the Brain.

  2016cites this paper
• Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.

  2016cites this paper
• Non-Viral Nucleic Acid Delivery Strategies to the Central Nervous System

  2016cites this paper
• Stereotaxic Surgical Targeting of the Nonhuman Primate Caudate and Putamen: Gene Therapy for Huntington's Disease.

  2016cites this paper
• Intraparenchymal ultrasound application and improved distribution of infusate with convection-enhanced delivery in rodent and nonhuman primate brain.

  2016cites this paper
• Concepts, technologies, and practices for drug delivery past the blood-brain barrier to the central nervous system.

  2016cites this paper
• Silencing of genes responsible for polyQ diseases using chemically modified single-stranded siRNAs.

  2016cites this paper
• Synthetic Polymer and Microbubble Systems for Gene Delivery to the Brain

  2016cites this paper
• Delivering a disease-modifying treatment for Huntington's disease.

  2015cites this paper
• MicroRNAs in CAG trinucleotide repeat expansion disorders: an integrated review of the literature.

  2015cites this paper
• Device‐Enabled Drug Infusion Therapies

  2015cites this paper
• Local Delivery of Therapeutic Nanoparticles for Treatment of Malignant Glioma

  2015cites this paper
• Continuous intraputamenal convection-enhanced delivery in adult rhesus macaques.

  2015cites this paper
• A holistic approach to targeting disease with polymeric nanoparticles

  2015cites this paper
• Therapeutic Strategies for Huntington's Disease.

  2015cites this paper
• Highly PEGylated DNA Nanoparticles Provide Uniform and Widespread Gene Transfer in the Brain

  2015cites this paper
• [Huntington's disease].

  2015cites this paper
• Onset Time and Durability of Huntingtin Suppression in Rhesus Putamen After Direct Infusion of Antihuntingtin siRNA

  2015cites this paper
• Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain

  2015cites this paper
• Huntingtin‐lowering strategies in Huntington's disease: Antisense oligonucleotides, small RNAs, and gene editing

  2014cites this paper
• Cell-based therapies for Huntington's disease.

  2014cites this paper
• Targets for future clinical trials in Huntington's disease: What's in the pipeline?

  2014cites this paper
• Huntington’s Disease: Molecular Pathogenesis and New Therapeutic Perspectives

  2014cites this paper
• Novel therapeutic strategies in major depression: focus on RNAi and ketamine.

  2014cites this paper
• Novel Central Nervous System Drug Delivery Systems

  2014cites this paper
• Convection‐Enhanced Drug Delivery to the Brain: Therapeutic Potential and Neuropathological Considerations

  2014cites this paper
• Oligonucleotide-based therapy for neurodegenerative diseases.

  2014cites this paper
• Oligonucleotide-based strategies to combat polyglutamine diseases

  2014cites this paper
• Stem Cell Treatments for Huntington’s Disease

  2014cites this paper
• The Study of Two Strategies for Decreasing Mutant Huntingtin: Degradation by Puromycin SensitiveAminoPeptidase and RNA Interference: A Dissertation

  2013cites this paper
• Gene therapies for spinocerebellar ataxia type 1

  2013cites this paper
• Huntington’s Enrichment Research Optimisation scheme AN INVESTIGATION INTO THE EFFECTS OF ENVIRONMENTAL ENRICHMENT ON CLINICAL MEASURES OF DISEASE PROGRESSION AND QUALITY OF LIFE IN PATIENTS WITH HUNTINGTON’S DISEASE

  2013cites this paper
• Huntington’s Disease: The Past, Present, and Future Search for Disease Modifiers

  2013cites this paper
• Self-assembling modified β-cyclodextrin nanoparticles as neuronal siRNA delivery vectors: focus on Huntington's disease.

  2013cites this paper
• Structure and mechanism of kynurenine 3-monooxygenase, a candidate huntington’s disease drug target

  2013influential citation
• Novel siRNA delivery strategy: a new “strand” in CNS translational medicine?

  2013cites this paper
• Recent Advances in RNA Interference Therapeutics for CNS Diseases

  2013cites this paper
• Gene Therapy for Misfolding Protein Diseases of the Central Nervous System

  2013cites this paper
• Technologies for Investigating the Physiological Barriers to Efficient Lipid Nanoparticle–siRNA Delivery

  2013cites this paper
• Non-viral nanosystems for gene and small interfering RNA delivery to the central nervous system: formulating the solution.

  2013cites this paper
• Emerging role of non-coding RNA in neural plasticity, cognitive function, and neuropsychiatric disorders

  2012cites this paper
• Huntington's disease: fighting on many fronts.

  2012cites this paper
• How can human pluripotent stem cells help decipher and cure Huntington's disease?

  2012cites this paper
• Increased brain radioactivity by intranasal 32P-labeled siRNA dendriplexes within in situ-forming mucoadhesive gels

  2012cites this paper
• Therapeutic approaches to preventing cell death in Huntington disease.

  2012cites this paper
• NEWS FROM THE SOCIETY FOR NEUROSCIENCE ANNUAL MEETING: Mutant Huntingtin Protein Suppressed in Animal Model of Huntingtonʼs Disease

  2012cites this paper
• An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases

  2012cites this paper
• Therapeutic Silencing of Mutant Huntingtin by Targeting Single Nucleotide Polymorphisms: A Dissertation

  2012cites this paper
• Mechanism of allele-selective inhibition of huntingtin expression by duplex RNAs that target CAG repeats: function through the RNAi pathway

  2012cites this paper
• Focused ultrasound for targeted delivery of siRNA and efficient knockdown of Htt expression.

  2012cites this paper
• Huntington disease

  2010cites this paper
• Disease: Understanding Its Novel Drugs and Treatments

  year unknowncites this paper