Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I. (Q30499512)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21123810%20AND%20SRC:MED&resulttype=core&format=json

17 January 2020

title

Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I (English)

1 reference

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17 January 2020

main subject

mucopolysaccharidosis I

1 reference

2

1 reference

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17 January 2020

Elizabeth Snella

10

1 reference

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17 January 2020

author name string

Ashley D Dierenfeld

1

1 reference

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17 January 2020

Carole A Vogler

3

1 reference

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17 January 2020

Charles H Vite

4

1 reference

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17 January 2020

Agnes H Chen

5

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17 January 2020

Merry Passage

6

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17 January 2020

Steven Le

7

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17 January 2020

Sahil Shah

8

1 reference

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17 January 2020

Jackie K Jens

9

1 reference

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17 January 2020

Karen L Kline

11

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17 January 2020

Jennifer D Parkes

12

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17 January 2020

Wendy A Ware

13

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17 January 2020

Lori E Moran

14

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17 January 2020

Amanda J Fales-Williams

15

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17 January 2020

Jane A Wengert

16

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17 January 2020

R David Whitley

17

1 reference

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17 January 2020

Daniel M Betts

18

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17 January 2020

Amy M Boal

19

1 reference

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17 January 2020

Elizabeth A Riedesel

20

1 reference

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17 January 2020

William Gross

21

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17 January 2020

N Matthew Ellinwood

22

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17 January 2020

Patricia I Dickson

23

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17 January 2020

language of work or name

English

0 references

publication date

1 December 2010

1 reference

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Science Translational Medicine

17 January 2020

published in

1 reference

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17 January 2020

volume

2

1 reference

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17 January 2020

page(s)

60ra89

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17 January 2020

issue

60

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Inadvertent propagation of factor VII deficiency in a canine mucopolysaccharidosis type I research breeding colony

17 January 2020

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1 reference

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Tandem mass spectrometry for the direct assay of lysosomal enzymes in dried blood spots: application to screening newborns for mucopolysaccharidosis I

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Radiographic evaluation of bones and joints in mucopolysaccharidosis I and VII dogs after neonatal gene therapy

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Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.

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Enzyme replacement therapy results in substantial improvements in early clinical phenotype in a mouse model of globoid cell leukodystrophy

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20 June 2018

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20 June 2018

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20 June 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice

26 November 2018

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26 November 2018

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26 November 2018

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Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I

26 November 2018

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12 December 2020

Meningiomas in young cats with mucopolysaccharidosis I

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12 December 2020

Radiographic findings in a canine model of mucopolysaccharidosis I. Changes associated with bone marrow transplantation

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12 December 2020

Tandem mass spectrometric analysis of dried blood spots for screening of mucopolysaccharidosis I in newborns

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12 December 2020

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Transplanted ER-MP12hi20-58med/hi myeloid progenitors produce resident macrophages from marrow that are therapeutic for lysosomal storage disease

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12 December 2020

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1 reference

12 December 2020

10.1126/SCITRANSLMED.3001380

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21123810%20AND%20SRC:MED&resulttype=core&format=json

17 January 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:21123810%20AND%20SRC:MED&resulttype=core&format=json

17 January 2020

1 reference