Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration.

No abstract is available for this paper.

• Publication year

  2012

• Venue

  Molecular Genetics and Metabolism

• Publication date

  2012-05-01

• Fields of study

  Medicine

• Identifiers
  DOI 10.1016/j.ymgme.2012.02.006PMID 22420937
• 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.

• Biodistribution and pharmacodynamics of recombinant human alpha-L-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations.

  2011cited by this paper
• Therapeutic Efficacy of Intranasally Delivered Mesenchymal Stem Cells in a Rat Model of Parkinson Disease

  2011cited by this paper
• Intranasal nerve growth factor enhances striatal neurogenesis in adult rats with focal cerebral ischemia

  2011cited by this paper
• Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I.

  2011influential reference
• Transnasal delivery of methotrexate to brain tumors in rats: a new strategy for brain tumor chemotherapy.

  2010cited by this paper
• Intranasal delivery to the central nervous system: mechanisms and experimental considerations.

  2010cited by this paper
• Intranasal drug targeting of hypocretin-1 (orexin-A) to the central nervous system.

  2009cited by this paper
• Intranasal delivery of cells to the brain.

  2009cited by this paper
• Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon system.

  2009cited by this paper
• Delivery of nerve growth factor to brain via intranasal administration and enhancement of brain uptake.

  2009cited by this paper
• A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I.

  2009cited by this paper
• Delivery of NGF to the Brain: Intranasal versus Ocular Administration in Anti-NGF Transgenic Mice

  2009cited by this paper
• Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression

  2008cited by this paper
• Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid.

  2007cited by this paper
• Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy.

  2007cited by this paper
• Intranasal administration of the growth-compromised HSV-2 vector DeltaRR prevents kainate-induced seizures and neuronal loss in rats and mice.

  2006cited by this paper
• Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.

  2005cited by this paper
• Intranasal delivery of nerve growth factor to protect the central nervous system against acute cerebral infarction.

  2004cited by this paper
• Intranasal administration of NAP, a neuroprotective peptide, decreases anxiety-like behavior in aging mice in the elevated plus maze.

  2004cited by this paper
• Enhanced Mucosal Immunoglobulin A Response of Intranasal Adenoviral Vector Human Immunodeficiency Virus Vaccine and Localization in the Central Nervous System

  2003cited by this paper
• Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB

  2003cited by this paper
• Effect of intranasal administration of Semliki Forest virus recombinant particles expressing reporter and cytokine genes on the progression of experimental autoimmune encephalomyelitis.

  2003cited by this paper
• Sniffing neuropeptides: a transnasal approach to the human brain

  2002cited by this paper
• Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I.

  1996cited by this paper
• Gene delivery into the central nervous system by nasal instillation in rats.

  1995cited by this paper

• Intranasal versus intravenous AAV delivery: A comparative analysis of brain-targeting efficiency and peripheral exposure in mice.

  2025cites this paper
• Intranasal insulin for the treatment of alcohol use disorder: design and methodology of an alcohol interaction randomized controlled trial

  2025cites this paper
• Intranasal delivery of drugs to the central nervous system of adult zebrafish

  2025cites this paper
• Adeno-associated virus vector delivery to the brain: Technology advancements and clinical applications.

  2024cites this paper
• Nanotechnology for enhanced nose-to-brain drug delivery in treating neurological diseases.

  2024cites this paper
• Exploring the Potential of Nasal Drug Delivery for Brain Targeted Therapy: A Detailed Analysis

  2024cites this paper
• The potential of gene delivery for the treatment of traumatic brain injury

  2024cites this paper
• AAV-BDNF gene therapy ameliorates a hypothalamic neuroinflammatory signature in the Magel2-null model of Prader-Willi syndrome

  2023cites this paper
• Mucopolysaccharidoses and the blood–brain barrier

  2022cites this paper
• Targeting the Central Nervous System in Lysosomal Storage Diseases: Strategies to Deliver Therapeutics Across the Blood-Brain Barrier.

  2022cites this paper
• Routes of administration for adeno-associated viruses carrying gene therapies for brain diseases

  2022cites this paper
• Administration of intramuscular AAV-BDNF and intranasal AAV-TrkB promotes neurological recovery via enhancing corticospinal synaptic connections in stroke rats.

  2022cites this paper
• A comprehensive review on carrier mediated nose to brain targeting: emphasis on molecular targets, current trends, future prospects, and challenges

  2022cites this paper
• Incisionless targeted adeno-associated viral vector delivery to the brain by focused ultrasound-mediated intranasal administration

  2022cites this paper
• Intranasal Application of Adeno-Associated Viruses: A Systematic Review.

  2022cites this paper
• Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I

  2021cites this paper
• Recent progress in nanocarriers for direct nose to brain drug delivery

  2021cites this paper
• NOSE-TO-BRAIN DRUG DELIVERY: AN UPDATE TO THE ALTERNATIVE PATH TO SUCCESSFUL TARGETED ANTI-MIGRAINE DRUGS

  2021cites this paper
• Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases

  2020cites this paper
• Engineered AAV8 capsid acquires heparin and AVB sepharose binding capacity but has altered in vivo transduction efficiency

  2020cites this paper
• Intranasal Insulin: a Treatment Strategy for Addiction

  2020cites this paper
• Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality.

  2019cites this paper
• Systemic Gene Delivery by Single-Dose Intracardiac Administration of scAAV2/9 and scAAV2/rh10 Variants in Newborn Rats

  2018cites this paper
• A New Perspective in Utilizing MMP-9 as a Therapeutic Target for Alzheimer’s Disease and Type 2 Diabetes Mellitus

  2018cites this paper
• Exosomes as Potential Transport Vehicles of Tetrahydrobiopterin, 6-Pyrovyoltetrahydrobiopterin-Synthase and Tripeptidyl-Peptidase I

  2018influential citation
• Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I

  2017influential citation
• Challenges of the Nose-to-Brain Route

  2017cites this paper
• Prolonged Expression of Secreted Enzymes in Dogs After Liver-Directed Delivery of Sleeping Beauty Transposons: Implications for Non-Viral Gene Therapy of Systemic Disease

  2017cites this paper
• Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives

  2017cites this paper
• Viral Vector Gene Delivery to the Brain for Treating Neurogenetic Diseases

  2017cites this paper
• Intranasal Administration of a Naked Plasmid Reached Brain Cells and Expressed Green Fluorescent Protein, a Candidate for Future Gene Therapy Studies.

  2017cites this paper
• Drug and Gene Delivery to the Central Nervous System for Neuroprotection

  2017influential citation
• Guanidinylated Neomycin Conjugation Enhances Intranasal Enzyme Replacement in the Brain.

  2017cites this paper
• Improving drug delivery technology for treating neurodegenerative diseases

  2016cites this paper
• Methods of Drug Delivery in Neurotrauma.

  2016cites this paper
• Rapid intranasal delivery of chloramphenicol acetyltransferase in the active form to different brain regions as a model for enzyme therapy in the CNS.

  2016cites this paper
• Perillyl Alcohol and Its Drug-Conjugated Derivatives as Potential Novel Methods of Treating Brain Metastases

  2016cites this paper
• Formulation and device design to increase nose to brain drug delivery

  2016cites this paper
• Intranasal Delivery of Recombinant AAV Containing BDNF Fused with HA2TAT: a Potential Promising Therapy Strategy for Major Depressive Disorder

  2016cites this paper
• Cell proliferation and metal accumulation in the subventricular zone and olfactory bulb in Parkinson’s disease

  2016cites this paper
• Intranasal gene delivery for treating Parkinson’s disease: overcoming the blood–brain barrier

  2015cites this paper
• Advances in Neurotherapeutic Delivery Technologies

  2015cites this paper
• BBB-targeting, protein-based nanomedicines for drug and nucleic acid delivery to the CNS.

  2015cites this paper
• The Olfactory System as a Route of Delivery for Agents to the Brain and Circulation

  2015cites this paper
• Transport Mechanisms of Neurotherapeutics across the Blood-Brain Barrier eBooks

  2015cites this paper
• High-dose enzyme replacement therapy in murine Hurler syndrome.

  2014cites this paper
• Standardization of α-L-iduronidase enzyme assay with Michaelis-Menten kinetics.

  2014cites this paper
• Inhibition of Let7c MicroRNA Is Neuroprotective in a Rat Intracerebral Hemorrhage Model

  2014cites this paper
• Systemic gene delivery to the central nervous system using Adeno-associated virus

  2014cites this paper
• Toward the successful delivery of lysosomal enzymes across the blood–brain barrier

  2013cites this paper
• Intranasal administration of oxytocin: Behavioral and clinical effects, a review

  2013cites this paper
• Enzyme Replacement in Neuronal Storage Disorders in the Pediatric Population

  2013cites this paper
• Influence of polymeric microcarriers on the in vivo intranasal uptake of an anti-migraine drug for brain targeting.

  2013cites this paper
• Direct nose to brain drug delivery via integrated nerve pathways bypassing the blood–brain barrier: an excellent platform for brain targeting

  2013cites this paper
• Animal models for lysosomal storage disorders

  2013cites this paper
• The Application of Clinical Genetics Dovepress Recent Advances in Gene Therapy for Lysosomal Storage Disorders

  year unknowncites this paper