(Q44016335)

29 November 2017

In vitro gene therapy of mucopolysaccharidosis type I by lentiviral vectors (English)

1 reference

29 November 2017

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mucopolysaccharidosis I

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based on heuristic

inferred from title

author

Angelo Leone Lombardo

series ordinal

4

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29 November 2017

5

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29 November 2017

6

Luigi Naldini

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29 November 2017

Paola Di Natale

1

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29 November 2017

Carmela Di Domenico

series ordinal

2

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29 November 2017

Guglielmo R D Villani

series ordinal

3

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29 November 2017

language of work or name

English

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publication date

1 June 2002

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29 November 2017

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29 November 2017

269

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29 November 2017

2764-2771

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29 November 2017

11

1 reference

Human alpha-L-iduronidase: cDNA isolation and expression

29 November 2017

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Molecular genetics of mucopolysaccharidosis type I: diagnostic, clinical, and biological implications.

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Mutational analysis of 85 mucopolysaccharidosis type I families: frequency of known mutations, identification of 17 novel mutations and in vitro expression of missense mutations.

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Enzyme-replacement therapy in mucopolysaccharidosis I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Retrovirus-mediated transfer of the human alpha-L-iduronidase cDNA into human hematopoietic progenitor cells leads to correction in trans of Hurler fibroblasts

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Uptake of alpha-(L)-iduronidase produced by retrovirally transduced fibroblasts into neuronal and glial cells in vitro

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Retroviral vector design studies toward hematopoietic stem cell gene therapy for mucopolysaccharidosis type I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Enzymatic correction and cross-correction of mucopolysaccharidosis type I fibroblasts by adeno-associated virus-mediated transduction of the alpha-L-iduronidase gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

The biogenesis of lysosomes

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

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/10.1046%2FJ.1432-1033.2002.02951.X

Murine mucopolysaccharidosis type I: targeted disruption of the murine alpha-L-iduronidase gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer.

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

A fluorometric assay using 4-methylumbelliferyl α-l-iduronide for the estimation of α-l-iduronidase activity and the detection of Hurler and Scheie syndromes

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Overexpression of the human lysosomal enzyme alpha-L-iduronidase in Chinese hamster ovary cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Characterization of novel safe lentiviral vectors derived from simian immunodeficiency virus (SIVmac251) that efficiently transduce mature human dendritic cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Pseudotyped human lentiviral vector-mediated gene transfer to airway epithelia in vivo.

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Clinical gene therapy in hematology: past and future

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Transduction of liver cells by lentiviral vectors: analysis in living animals by fluorescence imaging.

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

Mutations among Italian mucopolysaccharidosis type I patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

21 January 2018

1 reference

https://api.crossref.org/works/10.1046%2FJ.1432-1033.2002.02951.X

10.1046/J.1432-1033.2002.02951.X

21 January 2018

Identifiers

DOI

1 reference

29 November 2017

1 reference