(Q30519560)

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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

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scholarly article

1 reference

Europe PubMed Central

12 August 2017

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 (English)

1 reference

Europe PubMed Central

12 August 2017

0 references

lysosomal storage disease

1 reference

based on heuristic

inferred from title

author name string

Yong Hong Chen

1

1 reference

Europe PubMed Central

12 August 2017

Kristin Claflin

2

1 reference

Europe PubMed Central

12 August 2017

James C Geoghegan

3

1 reference

Europe PubMed Central

12 August 2017

Beverly L Davidson

4

1 reference

Europe PubMed Central

12 August 2017

language of work or name

1 reference

based on heuristic

inferred from title

publication date

15 May 2012

1 reference

Europe PubMed Central

12 August 2017

Molecular Therapy

1 reference

Europe PubMed Central

12 August 2017

20

1 reference

Europe PubMed Central

12 August 2017

7

1 reference

Europe PubMed Central

12 August 2017

1393-1399

1 reference

Europe PubMed Central

12 August 2017

Neurovascular pathways to neurodegeneration in Alzheimer's disease and other disorders

1 reference

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20 June 2018

Novel random peptide libraries displayed on AAV serotype 9 for selection of endothelial cell-directed gene transfer vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Peptide ligands incorporated into the threefold spike capsid domain to re-direct gene transduction of AAV8 and AAV9 in vivo

1 reference

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Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders

1 reference

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20 June 2018

Large-volume intrathecal enzyme delivery increases survival of a mouse model of late infantile neuronal ceroid lipofuscinosis

1 reference

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20 June 2018

The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice

1 reference

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20 June 2018

Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates

1 reference

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20 June 2018

Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery

1 reference

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20 June 2018

A peptide found by phage display discriminates a specific structure of a trisaccharide in heparin.

1 reference

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20 June 2018

Engaging neuroscience to advance translational research in brain barrier biology.

1 reference

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20 June 2018

Terminal N-linked galactose is the primary receptor for adeno-associated virus 9.

1 reference

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20 June 2018

Differential distribution of heparan sulfate glycoforms and elevated expression of heparan sulfate biosynthetic enzyme genes in the brain of mucopolysaccharidosis IIIB mice

1 reference

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20 June 2018

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

1 reference

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20 June 2018

Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy

1 reference

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20 June 2018

Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Recent developments in adeno-associated virus vector technology

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Novel challenges in exploring peptide ligands and corresponding tissue-specific endothelial receptors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Vascular targeting: recent advances and therapeutic perspectives

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Gene therapy for lysosomal storage diseases.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Vascular bed-targeted in vivo gene delivery using tropism-modified adeno-associated viruses

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Culture of murine brain microvascular endothelial cells that maintain expression and cytoskeletal association of tight junction-associated proteins

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Enzyme replacement therapy for the mucopolysaccharide storage disorders

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Steps toward mapping the human vasculature by phage display.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

A DNA-binding peptide from a phage display library

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Molecular addresses in blood vessels as targets for therapy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Incorporation of tumor-targeting peptides into recombinant adeno-associated virus capsids

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

NG2 proteoglycan-binding peptides target tumor neovasculature

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Molecular heterogeneity of the vascular endothelium revealed by in vivo phage display

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Molecular mechanism of lysosomal sialidase deficiency in galactosialidosis involves its rapid degradation

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis

1 reference

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20 June 2018

Defective sialic acid egress from isolated fibroblast lysosomes of patients with Salla disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

20 June 2018

Promising CNS-directed enzyme replacement therapy for lysosomal storage diseases

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

26 November 2018

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

26 November 2018

Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

26 November 2018

Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

26 November 2018

Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

26 November 2018

Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3392983

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Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII.

1 reference

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Hurler and Hunter syndromes: mutual correction of the defect in cultured fibroblasts

1 reference

https://pubmed.ncbi.nlm.nih.gov/22588273

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Defective lysosomal egress of free sialic acid (N-acetylneuraminic acid) in fibroblasts of patients with infantile free sialic acid storage disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/22588273

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Structure, function and metabolism of sialic acids

1 reference

https://pubmed.ncbi.nlm.nih.gov/22588273

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

The thiobarbituric acid assay of sialic acids

1 reference

https://pubmed.ncbi.nlm.nih.gov/22588273

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1038/MT.2012.100

1 reference

Europe PubMed Central

12 August 2017

1 reference

Europe PubMed Central

12 August 2017

1 reference

Europe PubMed Central

12 August 2017

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