Sialic acid deposition impairs the utility of AAV9, but not peptide-modified AAVs for brain gene therapy in a mouse model of lysosomal storage disease
about
Biology of adeno-associated viral vectors in the central nervous systemTherapies for the bone in mucopolysaccharidosesSystemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan.Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.Chondroitin Sulfate is the Primary Receptor for a Peptide-Modified AAV That Targets Brain Vascular Endothelium In Vivo.Therapies of mucopolysaccharidosis IVA (Morquio A syndrome).Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy.Gene therapy for neurologic manifestations of mucopolysaccharidoses.Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase.Methods for gene transfer to the central nervous system.Histochemical localization of palmitoyl protein thioesterase-1 activityMucopolysaccharidosis IIIB confers enhanced neonatal intracranial transduction by AAV8 but not by 5, 9 or rh10Blood-brain barrier structure and function and the challenges for CNS drug delivery.Gene delivery strategies for the treatment of mucopolysaccharidoses.Gene therapy for the neurological manifestations in lysosomal storage disorders.Central nervous system delivery of helper-dependent canine adenovirus corrects neuropathology and behavior in mucopolysaccharidosis type VII mice.Recent advances in gene therapy for lysosomal storage disorders.Emerging drugs for the treatment of mucopolysaccharidoses.Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis.Gene therapy for Mucopolysaccharidoses.Overcoming Limitations Inherent in Sulfamidase to Improve Mucopolysaccharidosis IIIA Gene Therapy.
P2860
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P2860
Sialic acid deposition impairs the utility of AAV9, but not peptide-modified AAVs for brain gene therapy in a mouse model of lysosomal storage disease
description
2012 nî lūn-bûn
@nan
2012 թուականի Մայիսին հրատարակուած գիտական յօդուած
@hyw
2012 թվականի մայիսին հրատարակված գիտական հոդված
@hy
2012年の論文
@ja
2012年論文
@yue
2012年論文
@zh-hant
2012年論文
@zh-hk
2012年論文
@zh-mo
2012年論文
@zh-tw
2012年论文
@wuu
name
Sialic acid deposition impairs ...... l of lysosomal storage disease
@ast
Sialic acid deposition impairs ...... l of lysosomal storage disease
@en
type
label
Sialic acid deposition impairs ...... l of lysosomal storage disease
@ast
Sialic acid deposition impairs ...... l of lysosomal storage disease
@en
prefLabel
Sialic acid deposition impairs ...... l of lysosomal storage disease
@ast
Sialic acid deposition impairs ...... l of lysosomal storage disease
@en
P2093
P2860
P356
P1433
P1476
Sialic acid deposition impairs ...... l of lysosomal storage disease
@en
P2093
Beverly L Davidson
James C Geoghegan
Kristin Claflin
Yong Hong Chen
P2860
P304
P356
10.1038/MT.2012.100
P577
2012-05-15T00:00:00Z