Long-term in vitro correction of alpha-L-iduronidase deficiency (Hurler syndrome) in human bone marrow. (Q37708615)

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17 October 2019

title

Long-term in vitro correction of alpha-L-iduronidase deficiency (Hurler syndrome) in human bone marrow (English)

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17 October 2019

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R Holt

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J J Hopwood

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E Spooncer

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17 October 2019

author name string

L J Fairbairn

1

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L S Lashford

2

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R H McDermott

4

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G Lebens

5

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17 October 2019

J E Arrand

6

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J R Arrand

7

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C E Hatton

10

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A Cooper

11

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G T Besley

12

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J E Wraith

13

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D S Anson

14

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T M Dexter

16

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17 October 2019

language of work or name

English

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publication date

1 March 1996

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

17 October 2019

published in

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17 October 2019

volume

93

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issue

5

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17 October 2019

page(s)

2025-2030

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Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts

17 October 2019

cites work

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Metabolic correction and cross-correction of mucopolysaccharidosis type II (Hunter syndrome) by retroviral-mediated gene transfer and expression of human iduronate-2-sulfatase

23 August 2017

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Enzyme replacement in a canine model of Hurler syndrome

23 August 2017

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23 August 2017

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Improved retroviral vectors for gene transfer and expression

23 August 2017

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Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection

23 August 2017

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Functional characterization of individual human hematopoietic stem cells cultured at limiting dilution on supportive marrow stromal layers

23 August 2017

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Restoration of normal lysosomal function in mucopolysaccharidosis type VII cells by retroviral vector-mediated gene transfer

23 August 2017

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Human alpha-L-iduronidase: cDNA isolation and expression

23 August 2017

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Retroviral gene transfer to primitive normal and leukemic hematopoietic cells using clinically applicable procedures

23 August 2017

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Immunoquantification and enzyme kinetics of alpha-L-iduronidase in cultured fibroblasts from normal controls and mucopolysaccharidosis type I patients

23 August 2017

1 reference

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Stromal support enhances cell-free retroviral vector transduction of human bone marrow long-term culture-initiating cells

23 August 2017

1 reference

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Structure and sequence of the human alpha-L-iduronidase gene

23 August 2017

1 reference

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Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer

23 August 2017

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Retroviral-mediated transfer of the human glucocerebrosidase gene into cultured Gaucher bone marrow

23 August 2017

1 reference

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Detection of specific sequences among DNA fragments separated by gel electrophoresis

23 August 2017

1 reference

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The mucopolysaccharidoses. Diagnosis, molecular genetics and treatment.

23 August 2017

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Differential regulation of primitive human hematopoietic cells in long-term cultures maintained on genetically engineered murine stromal cells

6 September 2017

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Overexpression of N-acetylgalactosamine-4-sulphatase induces a multiple sulphatase deficiency in mucopolysaccharidosis-type-VI fibroblasts

28 September 2017

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Cells expressing human glucocerebrosidase from a retroviral vector repopulate macrophages and central nervous system microglia after murine bone marrow transplantation

23 June 2018

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Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation

23 June 2018

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Construction and use of a safe and efficient amphotropic packaging cell line

23 June 2018

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Correction of mucopolysaccharidosis type I fibroblasts by retroviral-mediated transfer of the human alpha-L-iduronidase gene

23 June 2018

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Long-term outcome of Hurler syndrome following bone marrow transplantation

23 June 2018

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Disappearance of lysosomal storage in spleen and liver of mucopolysaccharidosis VII mice after transplantation of genetically modified bone marrow cells.

1 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=39903

RECONSTITUTION OF HAEMOPOIETIC SYSTEM WITH AUTOLOGOUS MARROW TAKEN DURING RELAPSE OF ACUTE MYELOBLASTIC LEUKAEMIA AND GROWN IN LONG-TERM CULTURE

1 September 2018

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12 December 2020

The use of bone marrow cells grown in long-term culture for autologous bone marrow transplantation in acute myeloid leukaemia: an update

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12 December 2020

Proliferation of spleen colony forming units (CFU-S8, CFU-S13) and cells with marrow repopulating ability

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12 December 2020

Long-term clinical progress in bone marrow transplanted mucopolysaccharidosis type I patients with a defined genotype

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12 December 2020

Fluorimetric assay for prenatal detection of Hurler and Scheie homozygotes or heterozygotes

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12 December 2020

Identifiers

DOI

10.1073/PNAS.93.5.2025

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:8700879%20AND%20SRC:MED&resulttype=core&format=json

17 October 2019

0 references

1 reference

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17 October 2019

PubMed publication ID

8700879

1 reference