CNS-directed gene therapy for lysosomal storage diseases
about
Clarifying lysosomal storage diseasesTargeted Polymeric Nanoparticles for Brain Delivery of High Molecular Weight Molecules in Lysosomal Storage DisordersInduced pluripotent stem cell models of lysosomal storage disorders.Hemichannels in neurodegenerative diseases: is there a link to pathology?Therapeutic approaches for lysosomal storage diseasesLentivector integration sites in ependymal cells from a model of metachromatic leukodystrophy: non-B DNA as a new factor influencing integration.Differential distribution of heparan sulfate glycoforms and elevated expression of heparan sulfate biosynthetic enzyme genes in the brain of mucopolysaccharidosis IIIB mice.Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primatesNeural stem cell gene therapy ameliorates pathology and function in a mouse model of globoid cell leukodystrophy.Gene therapy approaches for lysosomal storage disease: next-generation treatment.Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type AA GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB.Strategies for delivery of therapeutics into the central nervous system for treatment of lysosomal storage disorders.New biotechnological and nanomedicine strategies for treatment of lysosomal storage disordersViral vector-mediated gene transfer for CNS disease.Emerging therapies for neurodegenerative lysosomal storage disorders - from concept to reality.Gene therapy approaches for lysosomal storage disorders, a good model for the treatment of mendelian diseases.Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives.Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.Serum global metabolomics profiling reveals profound metabolic impairments in patients with MPS IIIA and MPS IIIB.Bio-Plex immunoassay measuring the quantity of lysosomal N-acetylgalactosamine-6-sulfatase protein in dried blood spots for the screening of mucopolysaccharidosis IVA in newborn: a pilot study.Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery.Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy.Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates.Status of newborn screening and follow up investigations for Mucopolysaccharidoses I and II in Taiwan.
P2860
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P2860
CNS-directed gene therapy for lysosomal storage diseases
description
2008 nî lūn-bûn
@nan
2008年の論文
@ja
2008年論文
@yue
2008年論文
@zh-hant
2008年論文
@zh-hk
2008年論文
@zh-mo
2008年論文
@zh-tw
2008年论文
@wuu
2008年论文
@zh
2008年论文
@zh-cn
name
CNS-directed gene therapy for lysosomal storage diseases
@ast
CNS-directed gene therapy for lysosomal storage diseases
@en
type
label
CNS-directed gene therapy for lysosomal storage diseases
@ast
CNS-directed gene therapy for lysosomal storage diseases
@en
prefLabel
CNS-directed gene therapy for lysosomal storage diseases
@ast
CNS-directed gene therapy for lysosomal storage diseases
@en
P2860
P1433
P1476
CNS-directed gene therapy for lysosomal storage diseases
@en
P2093
Mark E Haskins
Mark S Sands
P2860
P356
10.1111/J.1651-2227.2008.00660.X
P407
P577
2008-04-01T00:00:00Z