Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease (Q34202095)

31 July 2017

Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease (English)

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31 July 2017

9

Edward H Schuchman

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31 July 2017

James C Dodge

1

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31 July 2017

Jennifer Clarke

2

1 reference

31 July 2017

Antonius Song

3

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31 July 2017

Jie Bu

4

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31 July 2017

Wendy Yang

5

1 reference

31 July 2017

Tatyana V Taksir

6

1 reference

31 July 2017

Denise Griffiths

7

1 reference

31 July 2017

Michael A Zhao

8

1 reference

31 July 2017

Seng H Cheng

10

1 reference

31 July 2017

Catherine R O'Riordan

11

1 reference

31 July 2017

Lamya S Shihabuddin

12

1 reference

31 July 2017

Marco A Passini

13

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31 July 2017

Gregory R Stewart

14

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31 July 2017

language of work or name

English

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publication date

21 November 2005

1 reference

Proceedings of the National Academy of Sciences of the United States of America

31 July 2017

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31 July 2017

102

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31 July 2017

49

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31 July 2017

17822-17827

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Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice

31 July 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1308930

A mouse model of classical late-infantile neuronal ceroid lipofuscinosis based on targeted disruption of the CLN2 gene results in a loss of tripeptidyl-peptidase I activity and progressive neurodegeneration

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1308930

Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-defici

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1308930

Viral vectors for gene delivery to the nervous system

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1308930

Intracerebral transplantation of mesenchymal stem cells into acid sphingomyelinase-deficient mice delays the onset of neurological abnormalities and extends their life span.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1308930

Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1308930

Scaleable chromatographic purification process for recombinant adeno-associated virus (rAAV).

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1308930

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

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1308930

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

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1308930

Acid sphingomyelinase deficient mice: a model of types A and B Niemann-Pick disease

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1308930

AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease

5 July 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0509062102

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/10.1073%2FPNAS.0509062102

Patterned cerebellar Purkinje cell death in a transgenic mouse model of Niemann Pick type A/B disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0509062102

Projections from the upper lumbar cord to the cerebellar nuclei in the rat, studied by anterograde axonal tracing.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0509062102

Projections from the lowest lumbar and sacral-caudal segments to the cerebellar nuclei in the rat, studied by anterograde axonal tracing.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0509062102

Projections from the thoracic cord to the cerebellar nuclei in the rat, studied by anterograde axonal tracing.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0509062102

Uncrossed and crossed projections from the upper cervical spinal cord to the cerebellar nuclei in the rat, studied by anterograde axonal tracing.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0509062102

Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0509062102

Intracerebral transplantation of adult mouse neural progenitor cells into the Niemann-Pick-A mouse leads to a marked decrease in lysosomal storage pathology.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0509062102

Acid sphingomyelinase-deficient macrophages have defective cholesterol trafficking and efflux

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0509062102

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/10.1073%2FPNAS.0509062102

Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0509062102

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/10.1073%2FPNAS.0509062102

Long-term correction of hyperphenylalaninemia by AAV-mediated gene transfer leads to behavioral recovery in phenylketonuria mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0509062102

Adenovirus-mediated gene transfer results in decreased lysosomal storage in brain and total correction in liver of aspartylglucosaminuria (AGU) mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0509062102

Recombinant AAV serotype 1 transduction efficiency and tropism in the murine brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0509062102

Adeno-associated virus vector-mediated transduction in the cat brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0509062102

Targeted retrograde gene delivery for neuronal protection

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/16301517

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

AAV8-mediated hepatic expression of acid sphingomyelinase corrects the metabolic defect in the visceral organs of a mouse model of Niemann-Pick disease

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/16301517

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1073/PNAS.0509062102

1 reference

31 July 2017

0 references

1 reference

31 July 2017

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