Effective cell and gene therapy in a murine model of Gaucher disease (Q33256447)

24 July 2017

Effective cell and gene therapy in a murine model of Gaucher disease (English)

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24 July 2017

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1 reference

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Ida Berglin Enquist

1

1 reference

24 July 2017

Eva Nilsson

2

1 reference

24 July 2017

Andreas Ooka

3

1 reference

24 July 2017

Jan-Eric Månsson

4

1 reference

24 July 2017

Karin Olsson

5

1 reference

24 July 2017

Mats Ehinger

6

1 reference

24 July 2017

Roscoe O Brady

7

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24 July 2017

Johan Richter

8

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24 July 2017

Stefan Karlsson

9

1 reference

24 July 2017

language of work or name

English

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publication date

5 September 2006

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

24 July 2017

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24 July 2017

103

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24 July 2017

37

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24 July 2017

13819-13824

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Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

24 July 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Enzyme replacement in Gaucher disease

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Gaucher disease: complexity in a "simple" disorder

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Viable mouse models of acid beta-glucosidase deficiency: the defect in Gaucher disease

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

TGF-beta signaling-deficient hematopoietic stem cells have normal self-renewal and regenerative ability in vivo despite increased proliferative capacity in vitro

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Chemical chaperones increase the cellular activity of N370S beta -glucosidase: a therapeutic strategy for Gaucher disease

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

A chimeric mouse model of Gaucher disease

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Systemic inflammation in glucocerebrosidase-deficient mice with minimal glucosylceramide storage

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Retroviral transfer of the glucocerebrosidase gene into CD34+ cells from patients with Gaucher disease: in vivo detection of transduced cells without myeloablation

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Gaucher's disease: the best laid schemes of mice and men.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Gaucher's disease: molecular, genetic and enzymological aspects

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Mice with type 2 and 3 Gaucher disease point mutations generated by a single insertion mutagenesis procedure

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Consequences of beta-glucocerebrosidase deficiency in epidermis. Ultrastructure and permeability barrier alterations in Gaucher disease

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Metaxin, a gene contiguous to both thrombospondin 3 and glucocerebrosidase, is required for embryonic development in the mouse: implications for Gaucher disease

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Ten years' experience of bone marrow transplantation for Gaucher disease.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Inducible gene targeting in mice

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Demonstration of a deficiency of glucocerebroside-cleaving enzyme in Gaucher's disease

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Rapidly progressive type III Gaucher disease: deterioration following partial splenectomy.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Correction of glucocerebrosidase deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with Gaucher disease

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Expression of human glucocerebrosidase in murine long-term bone marrow cultures after retroviral vector-mediated transfer.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

High levels of human glucocerebrosidase activity in macrophages of long-term reconstituted mice after retroviral infection of hematopoietic stem cells

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Gaucher disease: new molecular approaches to diagnosis and treatment

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Efficient transfer and sustained high expression of the human glucocerebrosidase gene in mice and their functional macrophages following transplantation of bone marrow transduced by a retroviral vector

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

A less costly regimen of alglucerase to treat Gaucher's disease.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1564262

Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis.

21 June 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0606016103

Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0606016103

Animal model of Gaucher's disease from targeted disruption of the mouse glucocerebrosidase gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0606016103

Demonstration of feasibility of in vivo gene therapy for Gaucher disease using a chemically induced mouse model

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0606016103

Induced disruption of the transforming growth factor beta type II receptor gene in mice causes a lethal inflammatory disorder that is transplantable

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0606016103

Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0606016103

Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0606016103

Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/16954197

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Plasma chitotriosidase activity in Gaucher disease patients who have been treated either by bone marrow transplantation or by enzyme replacement therapy with alglucerase

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/16954197

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Human acid beta-glucosidase. Use of conduritol B epoxide derivatives to investigate the catalytically active normal and Gaucher disease enzymes

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/16954197

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1073/PNAS.0606016103

1 reference

24 July 2017

0 references

1 reference

24 July 2017

1 reference