Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease (Q34449867)

2 August 2017

title

Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease (English)

1 reference

2 August 2017

0 references

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4

Marina Cavazzana

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Christof von Kalle

22

Christof von Kalle

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Edouard de Dreuzy

Edouard de Dreuzy

9

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Emmanuel Payen

Emmanuel Payen

23

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Philippe Leboulch

15

Philippe Leboulch

1 reference

2 August 2017

21

Manfred Schmidt

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2 August 2017

1

Olivier Negre

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2 August 2017

8

Maria Denaro

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2 August 2017

3

Yves Beuzard

1 reference

2 August 2017

Cynthia C Bartholomae

2

Cynthia Bartholomae

1 reference

2 August 2017

7

Annette Deichmann

1 reference

2 August 2017

Lauryn Christiansen

5

1 reference

2 August 2017

Céline Courne

6

1 reference

2 August 2017

Mitchell Finer

10

1 reference

2 August 2017

Raffaele Fronza

11

1 reference

2 August 2017

Béatrix Gillet-Legrand

12

1 reference

2 August 2017

Christophe Joubert

13

1 reference

2 August 2017

Robert Kutner

14

1 reference

2 August 2017

Leïla Maouche

16

1 reference

2 August 2017

Anaïs Paulard

17

1 reference

2 August 2017

Francis J Pierciey

18

1 reference

2 August 2017

Michael Rothe

19

1 reference

2 August 2017

Byoung Ryu

20

1 reference

2 August 2017

Gabor Veres

24

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2 August 2017

language of work or name

English

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publication date

1 January 2015

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2 August 2017

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2 August 2017

volume

15

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2 August 2017

issue

1

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2 August 2017

page(s)

64-81

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Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

2 August 2017

cites work

1 reference

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

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Advances in stem cell transplantation and gene therapy in the β-hemoglobinopathies.

6 July 2018

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Bioinformatic clonality analysis of next-generation sequencing-derived viral vector integration sites

6 July 2018

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Analyzing the number of common integration sites of viral vectors--new methods and computer programs

6 July 2018

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Development of novel efficient SIN vectors with improved safety features for Wiskott-Aldrich syndrome stem cell based gene therapy

6 July 2018

1 reference

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Correction of murine β-thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion

6 July 2018

1 reference

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Distribution of lentiviral vector integration sites in mice following therapeutic gene transfer to treat β-thalassemia

6 July 2018

1 reference

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Stem-cell gene therapy for the Wiskott-Aldrich syndrome

6 July 2018

1 reference

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

6 July 2018

1 reference

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High-dose cyclophosphamide for graft-versus-host disease prevention

6 July 2018

1 reference

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

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpression

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Complications and treatment of patients with β-thalassemia in France: results of the National Registry

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

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

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Gene specificity of suppression of transgene-mediated insertional transcriptional activation by the chicken HS4 insulator

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Production, concentration and titration of pseudotyped HIV-1-based lentiviral vectors

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Incorporating double copies of a chromatin insulator into lentiviral vectors results in less viral integrants

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Optimized lentiviral vector design improves titer and transgene expression of vectors containing the chicken beta-globin locus HS4 insulator element

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Simplified production and concentration of HIV-1-based lentiviral vectors using HYPERFlask vessels and anion exchange membrane chromatography

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Improved survival of thalassaemia major in the UK and relation to T2* cardiovascular magnetic resonance

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Role of PSIP1/LEDGF/p75 in lentiviral infectivity and integration targeting

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

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

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

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

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

A phase I/II clinical trial of beta-globin gene therapy for beta-thalassemia

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

A role for LEDGF/p75 in targeting HIV DNA integration

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Dynamics of transgene expression in a neural stem cell line transduced with lentiviral vectors incorporating the cHS4 insulator.

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

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

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Permanent and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

HIV-1 integration in the human genome favors active genes and local hotspots

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Position-effect protection and enhancer blocking by the chicken beta-globin insulator are separable activities

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Compendium of chemical carcinogens by target organ: results of chronic bioassays in rats, mice, hamsters, dogs, and monkeys

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Lymphomas and high-level expression of murine leukemia viruses in CFW mice.

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Leukemia and lymphoma incidence in rodents exposed to low-frequency magnetic fields

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

The protein CTCF is required for the enhancer blocking activity of vertebrate insulators

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Parallel assessment of globin lentiviral transfer in induced pluripotent stem cells and adult hematopoietic stem cells derived from the same transplanted β-thalassemia patient

6 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Lentivirus vectors in β-thalassemia.

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

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

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Prospective assessment of health-related quality of life in pediatric patients with beta-thalassemia following hematopoietic stem cell transplantation

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Progress in hematopoietic stem cell transplantation as allogeneic cellular gene therapy in thalassemia.

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

The life of patients with thalassemia major

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Comprehensive genomic access to vector integration in clinical gene therapy

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR).

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Importance of murine study design for testing toxicity of retroviral vectors in support of phase I trials.

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

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

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Revised guides for organ sampling and trimming in rats and mice--Part 2. A joint publication of the RITA and NACAD groups

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

Revised guides for organ sampling and trimming in rats and mice – Part 1

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4440358

A mouse model for β-thalassemia

27 October 2018

1 reference

12 December 2020

Mouse beta thalassemia, a model for the membrane defects of erythrocytes in the human disease

1 reference

12 December 2020

Lymphomas and leukemias in mice

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12 December 2020

Abstracts of the American Society of Gene & Cell Therapy (ASGGT) 16th Annual Meeting. May 15-18, 2013. Salt Lake City, Utah, USA

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12 December 2020

Marrow transplantation in patients with advanced thalassemia

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12 December 2020

Revised guides for organ sampling and trimming in rats and mice--Part 3. A joint publication of the RITA and NACAD groups

1 reference

12 December 2020

10.2174/1566523214666141127095336

Identifiers

DOI

1 reference

2 August 2017

1 reference

2 August 2017

1 reference