(Q34356914)

1 August 2017

title

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy (English)

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1 August 2017

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28

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1 August 2017

30

Christof von Kalle

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Luigi Naldini

37

Luigi Naldini

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Francesca Fumagalli

Francesca Fumagalli

5

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Andrea Calabria

Andrea Calabria

9

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Eugenio Montini

Eugenio Montini

2

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Sabata Martino

Sabata Martino

8

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Jaap J. Boelens

Jaap J Boelens

21

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Alessandro Aiuti

Alessandro Aiuti

35

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Maria Grazia Roncarolo

Maria Grazia Roncarolo

34

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Claudio Bordignon

31

Claudio Bordignon

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1 August 2017

16

Gianluigi Zanetti

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1 August 2017

24

Manfred Schmidt

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1 August 2017

32

Fabio Ciceri

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1 August 2017

Tiziana Plati

6

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Maria Sessa

Maria Sessa

36

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Clelia Di Serio

27

Clelia Di Serio

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1 August 2017

Alessandra Biffi

1

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1 August 2017

Laura Lorioli

3

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1 August 2017

Martina Cesani

4

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1 August 2017

Cristina Baldoli

7

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1 August 2017

Sabrina Canale

10

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1 August 2017

Fabrizio Benedicenti

11

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1 August 2017

Giuliana Vallanti

12

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1 August 2017

Luca Biasco

13

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1 August 2017

Simone Leo

14

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1 August 2017

Nabil Kabbara

15

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1 August 2017

William B Rizzo

17

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1 August 2017

Nalini A L Mehta

18

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1 August 2017

Maria Pia Cicalese

19

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1 August 2017

Miriam Casiraghi

20

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1 August 2017

Ubaldo Del Carro

22

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1 August 2017

David J Dow

23

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1 August 2017

Andrea Assanelli

25

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1 August 2017

Victor Neduva

26

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1 August 2017

Jason Gardner

29

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Attilio Rovelli

33

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1 August 2017

language of work or name

English

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publication date

11 July 2013

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1 August 2017

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1 August 2017

volume

341

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1 August 2017

issue

6148

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1 August 2017

page(s)

1233158

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Metachromatic leukodystrophy: an overview of current and prospective treatments

1 August 2017

cites work

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Gene therapy for leukodystrophies

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Microglia: The Effector Cell for Reconstitution of the Central Nervous System following Bone Marrow Transplantation for Lysosomal and Peroxisomal Storage Diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Hematopoietic cell transplantation activity in Europe for inherited metabolic diseases: open issues and future directions

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Current international perspectives on hematopoietic stem cell transplantation for inherited metabolic disorders.

21 January 2018

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https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Trends in haematopoietic cell transplantation for inborn errors of metabolism

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Gene therapy for severe combined immunodeficiency: are we there yet?

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Ex vivo gene transfer and correction for cell-based therapies

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Metachromatic leukodystrophy - mutation analysis provides further evidence of genotype-phenotype correlation

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Development and reliability of a classification system for gross motor function in children with metachromatic leucodystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Metachromatic leukodystrophy: natural course of cerebral MRI changes in relation to clinical course.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Real-time definition of non-randomness in the distribution of genomic events

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Fate mapping analysis reveals that adult microglia derive from primitive macrophages

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Targeting gene-modified hematopoietic cells to the central nervous system: use of green fluorescent protein uncovers microglial engraftment.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Localized donor cells in brain of a Hunter disease patient after cord blood stem cell transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Evidence for the cure of HIV infection by CCR5Δ32/Δ32 stem cell transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Physiological promoters reduce the genotoxic risk of integrating gene vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Concurrent and predictive validity of the cognitive adaptive test/clinical linguistic and auditory milestone scale (CAT/CLAMS) and the Mental Developmental Index of the Bayley Scales of Infant Development.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Sedation for children with metachromatic leukodystrophy undergoing MRI.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Expression and purification of a human, soluble Arylsulfatase A for Metachromatic Leukodystrophy enzyme replacement therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

RTCGD: retroviral tagged cancer gene database

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Detecting statistically significant common insertion sites in retroviral insertional mutagenesis screens

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Stem-cell gene therapy for the Wiskott-Aldrich syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

Comparison of human cord blood engraftment between immunocompromised mouse strains

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCIENCE.1233158

21 January 2018

Identifiers

DOI

10.1126/SCIENCE.1233158

1 reference

1 August 2017

1 reference