[go: up one dir, main page]
More Web Proxy on the site http://driver.im/

Beard et al., 2015 - Google Patents

Continual low-dose infusion of sulfamidase is superior to intermittent high-dose delivery in ameliorating neuropathology in the MPS IIIA mouse brain

Beard et al., 2015

View HTML
Document ID
4345145194590963679
Author
Beard H
Hassiotis S
Luck A
Rozaklis T
Hopwood J
Hemsley K
Publication year
Publication venue
JIMD Reports, Volume 29

External Links

Snippet

Abstract Mucopolysaccharidosis IIIA (MPS IIIA) is a neurodegenerative lysosomal storage disorder characterised by progressive loss of learned skills, sleep disturbance and behavioural problems. Reduced activity of lysosomal sulfamidase results in accumulation of …
Continue reading at www.ncbi.nlm.nih.gov (HTML) (other versions)

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine, rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine, rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
    • A61K49/00Preparations for testing in vivo
    • A61K49/0004Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/13Amines
    • A61K31/135Amines having aromatic rings, e.g. ketamine, nortriptyline
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/16Amides, e.g. hydroxamic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/30Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue

Similar Documents

Publication Publication Date Title
Heinz et al. Reduced central serotonin transporters in alcoholism
Muenzer et al. A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II
Vogler et al. Enzyme replacement in murine mucopolysaccharidosis type VII: neuronal and glial response to β-glucuronidase requires early initiation of enzyme replacement therapy
Rafi et al. Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease
Chen et al. Comparing routes of delivery for nanoliposomal irinotecan shows superior anti-tumor activity of local administration in treating intracranial glioblastoma xenografts
Vite et al. Effective gene therapy for an inherited CNS disease in a large animal model
Gill et al. Direct brain infusion of glial cell line–derived neurotrophic factor in Parkinson disease
Savas et al. Intracerebral injection of sulfamidase delays neuropathology in murine MPS-IIIA
US10080783B2 (en) Intraventricular enzyme delivery for lysosomal storage diseases
US7442372B2 (en) Delivery of therapeutic compounds to the brain and other tissues
Calias et al. CNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorder
Hemsley et al. Effect of high dose, repeated intra‐cerebrospinal fluid injection of sulphamidase on neuropathology in mucopolysaccharidosis type IIIA mice
Beard et al. Determination of the role of injection site on the efficacy of intra-CSF enzyme replacement therapy in MPS IIIA mice
Nagahara et al. MR-guided delivery of AAV2-BDNF into the entorhinal cortex of non-human primates
Xu et al. Large-volume intrathecal enzyme delivery increases survival of a mouse model of late infantile neuronal ceroid lipofuscinosis
Hemsley et al. Examination of intravenous and intra‐CSF protein delivery for treatment of neurological disease
Vegting et al. The effects of ecstasy on neurotransmitter systems: a review on the findings of molecular imaging studies
King et al. Low‐dose, continuous enzyme replacement therapy ameliorates brain pathology in the neurodegenerative lysosomal disorder mucopolysaccharidosis type IIIA
Marcó et al. Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal
Sohn et al. Improvement of CNS defects via continuous intrathecal enzyme replacement by osmotic pump in mucopolysaccharidosis type II mice
Xhima et al. Ultrasound delivery of a TrkA agonist confers neuroprotection to Alzheimer-associated pathologies
Huttunen et al. Intraputamenal cerebral dopamine neurotrophic factor in Parkinson's disease: a randomized, double‐blind, multicenter phase 1 trial
Banks et al. Effects of cocaine and MDMA self-administration on serotonin transporter availability in monkeys
Pienaar et al. The cellular diversity of the pedunculopontine nucleus: relevance to behavior in health and aspects of Parkinson’s disease
King et al. Evaluation of enzyme dose and dose-frequency in ameliorating substrate accumulation in MPS IIIA Huntaway dog brain