(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

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

17 January 2020

main subject

mucopolysaccharidosis I

1 reference

2

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

Elizabeth Snella

10

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

author name string

Ashley D Dierenfeld

1

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

Carole A Vogler

3

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

Charles H Vite

4

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

Agnes H Chen

5

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

Merry Passage

6

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

Steven Le

7

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

Sahil Shah

8

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

Jackie K Jens

9

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

Karen L Kline

11

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

Jennifer D Parkes

12

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

Wendy A Ware

13

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

Lori E Moran

14

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

Amanda J Fales-Williams

15

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

Jane A Wengert

16

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

R David Whitley

17

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

Daniel M Betts

18

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

Amy M Boal

19

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

Elizabeth A Riedesel

20

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

William Gross

21

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

N Matthew Ellinwood

22

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

Patricia I Dickson

23

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

language of work or name

English

0 references

publication date

1 December 2010

1 reference

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

Science Translational Medicine

17 January 2020

published in

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

volume

2

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

issue

60

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

page(s)

60ra89

1 reference

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

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

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=3075726

IDS crossing of the blood-brain barrier corrects CNS defects in MPSII mice

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Mannose 6-phosphate receptor-mediated transport of sulfamidase across the blood-brain barrier in the newborn mouse

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-L-iduronidase (laronidase).

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Bone marrow transplantation for feline mucopolysaccharidosis I.

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Efficacy of enzyme replacement therapy in alpha-mannosidosis mice: a preclinical animal study

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Successful induction of immune tolerance to enzyme replacement therapy in canine mucopolysaccharidosis I

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Enzyme replacement therapy in a mouse model of aspartylglycosaminuria

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Genotype-phenotype correlations in mucopolysaccharidosis type I using enzyme kinetics, immunoquantification and in vitro turnover studies.

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Enzyme replacement therapy for murine mucopolysaccharidosis type VII

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

A canine model of human alpha-L-iduronidase deficiency

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Corneal opacity in canine MPS I. Changes after bone marrow transplantation.

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Long-term effects of bone marrow transplantation in dogs with mucopolysaccharidosis I.

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Long-term outcome of Hurler syndrome following bone marrow transplantation.

20 June 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001380

Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001380

Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001380

A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001380

Enzyme-replacement therapy from birth delays the development of behavior and learning problems in mucopolysaccharidosis type IIIA mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001380

Replacement therapy in Mucopolysaccharidosis type VI: advantages of early onset of therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001380

Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001380

Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001380

Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001380

Early detection of Pompe disease by newborn screening is feasible: results from the Taiwan screening program.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001380

Simultaneous preparation and quantitation of proteoglycans by precipitation with alcian blue.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001380

Early administration of enzyme replacement therapy for Pompe disease: short-term follow-up results.

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

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

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Brain MRI findings in patients with mucopolysaccharidosis types I and II and mild clinical presentation.

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

Enzyme replacement in murine mucopolysaccharidosis type VII: neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy.

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3075726

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

26 November 2018

1 reference

12 December 2020

Meningiomas in young cats with mucopolysaccharidosis I

1 reference

12 December 2020

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

1 reference

12 December 2020

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

1 reference

12 December 2020

Long-term outcomes of adaptive functions for children with mucopolysaccharidosis I (Hurler syndrome) treated with hematopoietic stem cell transplantation

1 reference

12 December 2020

Allogeneic bone marrow transplantation for lysosomal storage diseases. The European Group for Bone Marrow Transplantation

1 reference

12 December 2020

Hurler-like phenotype: enzymatic diagnosis in dried blood spots on filter paper

1 reference

12 December 2020

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

1 reference

12 December 2020

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

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