Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes. (Q34153746)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21139569%20AND%20SRC:MED&resulttype=core&format=json

17 January 2020

title

Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes (English)

1 reference

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17 January 2020

0 references

1 reference

2

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17 January 2020

Elizabeth Snella

7

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17 January 2020

Eman E A Mohammed

8

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17 January 2020

Christopher B Thomson

9

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17 January 2020

author name string

N Matthew Ellinwood

1

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17 January 2020

Nathalie Desmaris

3

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17 January 2020

Stéphanie Bigou

4

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17 January 2020

Song Liu

5

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17 January 2020

Jackie K Jens

6

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17 January 2020

Sylvie Raoul

10

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17 January 2020

Béatrice Joussemet

11

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17 January 2020

Françoise Roux

12

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17 January 2020

Yan Chérel

13

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17 January 2020

Yaouen Lajat

14

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17 January 2020

Monique Piraud

15

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17 January 2020

Rachid Benchaouir

16

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17 January 2020

Stephan Hermening

17

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17 January 2020

Harald Petry

18

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17 January 2020

Roseline Froissart

19

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17 January 2020

Marc Tardieu

20

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17 January 2020

Carine Ciron

21

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17 January 2020

Philippe Moullier

22

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17 January 2020

Jennifer Parkes

23

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17 January 2020

Karen L Kline

24

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17 January 2020

Irène Maire

25

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17 January 2020

Marie-Thérèse Vanier

26

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17 January 2020

Jean-Michel Heard

27

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17 January 2020

Marie-Anne Colle

28

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17 January 2020

publication date

7 December 2010

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17 January 2020

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17 January 2020

volume

19

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17 January 2020

issue

2

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17 January 2020

page(s)

251-259

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21139569%20AND%20SRC:MED&resulttype=core&format=json

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

17 January 2020

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Results from a phase I safety trial of hAADC gene therapy for Parkinson 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=3034858

Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Secondary accumulation of gangliosides in lysosomal storage disorders

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Gene therapy for lysosomal storage diseases: the lessons and promise of animal models

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Insect cells as a factory to produce adeno-associated virus type 2 vectors

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding alpha-N-acetylglucosaminidase

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Cloning and expression of the gene involved in Sanfilippo B syndrome (mucopolysaccharidosis III B)

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Overexpression of the human lysosomal enzyme alpha-L-iduronidase in Chinese hamster ovary cells

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Canine alpha-L-iduronidase deficiency. A model of mucopolysaccharidosis I

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

4-Methylumbelliferyl alpha-N-acetylglucosaminidase activity for diagnosis of Sanfilippo B 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=3034858

Two procedures to remove polar contaminants from a crude brain lipid extract by using prepacked reversed-phase columns

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Diagnostic test for mucopolysaccharidosis. I. Direct method for quantifying excessive urinary glycosaminoglycan excretion.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Human alpha-L-iduronidase: cDNA isolation and expression

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Architecture of the canine IDUA gene and mutation underlying canine mucopolysaccharidosis I.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Human alpha-iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: vector diffusion and biodistribution

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

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

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Immune responses to AAV in a phase I study for Canavan disease.

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

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

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Prevention of neuropathology in the mouse model of Hurler syndrome

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

A model of mucopolysaccharidosis IIIB (Sanfilippo syndrome type IIIB): N‐acetyl‐α‐D‐glucosaminidase deficiency in Schipperke dogs

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

Targeted disruption of the mouse sphingolipid activator protein gene: a complex phenotype, including severe leukodystrophy and wide-spread storage of multiple sphingolipids.

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3034858

The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I

28 October 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21139569

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/MT.2010.265

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21139569%20AND%20SRC:MED&resulttype=core&format=json

17 January 2020

1 reference

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17 January 2020

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