(Q30541387)

English

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.

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scholarly article

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Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model (English)

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

0 references

mucopolysaccharidosis I

1 reference

based on heuristic

inferred from title

1 reference

based on heuristic

inferred from title

Letterio S. Politi

3

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

7

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Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

13

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Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

14

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

Alessandro Rubinacci

17

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Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

Riccardo Brambilla

18

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

Angelo Quattrini

19

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

22

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Europe PubMed Central

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14 February 2020

author name string

Ilaria Visigalli

1

1 reference

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14 February 2020

Stefania Delai

2

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14 February 2020

Carmela Di Domenico

4

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

Federica Cerri

5

1 reference

Europe PubMed Central

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14 February 2020

Emanuela Mrak

6

1 reference

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14 February 2020

Daniela Ungaro

8

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

Merel Stok

9

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

Francesca Sanvito

10

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

Elisabetta Mariani

11

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

Lidia Staszewsky

12

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

Francesca Cecere

15

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

Ubaldo Del Carro

16

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

Paola Di Natale

20

1 reference

Europe PubMed Central

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14 February 2020

Katherine Ponder

21

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

Alessandra Biffi

23

1 reference

Europe PubMed Central

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14 February 2020

language of work or name

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publication date

16 September 2010

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Europe PubMed Central

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14 February 2020

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

116

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Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

24

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

5130-5139

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

Neonatal intravenous injection of a gammaretroviral vector has a low incidence of tumor induction in mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

A self-inactivating gamma-retroviral vector reduces manifestations of mucopolysaccharidosis I in mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

Early neurodegeneration progresses independently of microglial activation by heparan sulfate in the brain of mucopolysaccharidosis IIIB mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

Enzyme replacement improves nervous system pathology and function in a mouse model for metachromatic leukodystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

Cord-blood transplants from unrelated donors in patients with Hurler's syndrome

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

Molecular evidence of lentiviral vector-mediated gene transfer into human self-renewing, multi-potent, long-term NOD/SCID repopulating hematopoietic cells

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

Longevity, body weight, and neoplasia in ad libitum-fed and diet-restricted C57BL6 mice fed NIH-31 open formula diet

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

The mucopolysaccharidoses. Diagnosis, molecular genetics and treatment.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

20 June 2018

Modified sound-evoked brainstem potentials in Foxp2 mutant mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

26 November 2018

Heparin cofactor II–thrombin complex: A biomarker of MPS disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

26 November 2018

Safety of arylsulfatase A overexpression for gene therapy of metachromatic leukodystrophy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

26 November 2018

Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

26 November 2018

Effect of neonatal administration of a retroviral vector expressing alpha-L-iduronidase upon lysosomal storage in brain and other organs in mucopolysaccharidosis I mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

26 November 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

26 November 2018

Widespread correction of lysosomal storage following intrahepatic injection of a recombinant adeno-associated virus in the adult MPS VII mouse

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

26 November 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

26 November 2018

Advances in the treatment of mucopolysaccharidosis type I.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

26 November 2018

Development of a human adaptive immune system in cord blood cell-transplanted mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

26 November 2018

Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

26 November 2018

Distinct classes of human stem cells that differ in proliferative and self-renewal potential

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

26 November 2018

Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

26 November 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3709639

26 November 2018

Laronidase (Aldurazyme): enzyme replacement therapy for mucopolysaccharidosis type I

1 reference

https://pubmed.ncbi.nlm.nih.gov/20847202

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Evaluation of heparin cofactor II-thrombin complex as a biomarker on blood spots from mucopolysaccharidosis I, IIIA and IIIB mice

1 reference

https://pubmed.ncbi.nlm.nih.gov/20847202

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy

1 reference

https://pubmed.ncbi.nlm.nih.gov/20847202

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Transduction of Human CD34+CD38- Bone Marrow and Cord Blood-Derived SCID-Repopulating Cells with Third-Generation Lentiviral Vectors

1 reference

https://pubmed.ncbi.nlm.nih.gov/20847202

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1182/BLOOD-2010-04-278234

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

PMC publication ID

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

PubMed publication ID

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20847202%20AND%20SRC:MED&resulttype=core&format=json

14 February 2020

ResearchGate publication ID

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