CNS-directed gene therapy for lysosomal storage diseases (Q35923467)

11 August 2017

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title

CNS-directed gene therapy for lysosomal storage diseases (English)

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11 August 2017

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Mark S Sands

1

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11 August 2017

Mark E Haskins

series ordinal

2

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11 August 2017

language of work or name

English

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publication date

1 April 2008

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11 August 2017

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11 August 2017

97

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11 August 2017

457

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11 August 2017

22-27

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Metachromatic leukodystrophy: genetics, pathogenesis and therapeutic options

11 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

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

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Stem cell bone marrow transplantation in patients with metabolic storage diseases.

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Lifetime correction of genetic deficiency in mice with a single injection of helper-dependent adenoviral vector

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Engraftment of genetically engineered amniotic epithelial cells corrects lysosomal storage in multiple areas of the brain in mucopolysaccharidosis type VII mice

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system.

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

AAV vectors: is clinical success on the horizon?

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Prevalence of lysosomal storage disorders

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Efficient transduction of nondividing human cells by feline immunodeficiency virus lentiviral vectors

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of β-glucuronidase

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Adenovirus-mediated gene transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Bone marrow transplantation corrects the enzyme defect in neurons of the central nervous system in a lysosomal storage disease

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

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

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Lysosomal storage diseases

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Structure and function of the mannose 6-phosphate/insulinlike growth factor II receptors

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

The HIV Tat protein transduction domain improves the biodistribution of beta-glucuronidase expressed from recombinant viral vectors.

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Herpesvirus vector gene transfer and expression of beta-glucuronidase in the central nervous system of MPS VII mice

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Human beta-glucuronidase: in vivo clearance and in vitro uptake by a glycoprotein recognition system on reticuloendothelial cells

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Brain transplantation of genetically modified bone marrow stromal cells corrects CNS pathology and cognitive function in MPS VII mice

11 July 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Long-term expression of beta-glucuronidase by genetically modified human neural progenitor cells grafted into the mouse central nervous system.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Widespread distribution of beta-hexosaminidase activity in the brain of a Sandhoff mouse model after coinjection of adenoviral vector and mannitol.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Effective gene therapy for an inherited CNS disease in a large animal model

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

AAV vector integration sites in mouse hepatocellular carcinoma.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Significantly increased expression of beta-glucuronidase in the central nervous system of mucopolysaccharidosis type VII mice from the latency-associated transcript promoter in a nonpathogenic herpes simplex virus type 1 vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Gene therapy of the brain in the dog model of Hurler's syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Enhanced secretion and uptake of beta-glucuronidase improves adeno-associated viral-mediated gene therapy of mucopolysaccharidosis type VII mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Intraventricular administration of recombinant adenovirus to neonatal twitcher mouse leads to clinicopathological improvements

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

AAV-mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Central nervous system-directed AAV2/5-mediated gene therapy synergizes with bone marrow transplantation in the murine model of globoid-cell leukodystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111/J.1651-2227.2008.00660.X

Use of nonviral promoters in adenovirus-mediated gene therapy: reduction of lysosomal storage in the aspartylglucosaminuria mouse

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

CNS-directed AAV2-mediated gene therapy ameliorates functional deficits in a murine model of infantile neuronal ceroid lipofuscinosis.

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice.

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream.

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Extensive beta-glucuronidase activity in murine central nervous system after adenovirus-mediated gene transfer to brain

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3340572

Inborn errors of mucopolysaccharide metabolism

24 September 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/18339183

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1111/J.1651-2227.2008.00660.X

1 reference

11 August 2017

1 reference

11 August 2017

PubMed publication ID

18339183

1 reference