Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome (Q37089728)

18 August 2017

Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome (English)

1 reference

18 August 2017

0 references

30

Alain Fischer

0 references

Marina Cavazzana

33

Marina Cavazzana

0 references

Salima Hacein-Bey-Abina

1

Salima Hacein-Bey Abina

0 references

Jean-Marc Tréluyer

18

Jean-Marc Treluyer

0 references

Capucine Picard

7

Capucine Picard

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Laure Caccavelli

Laure Caccavelli

4

0 references

Sébastien Héritier

Sébastien Héritier

19

0 references

Emmanuelle M. Six

Emmanuelle Six

8

0 references

Fabien Touzot

Fabien Touzot

15

0 references

Fulvio Mavilio

Fulvio Mavilio

16

0 references

Adrian Thrasher

Adrian J Thrasher

31

0 references

Johanna Blondeau

3

Johanna Blondeau

1 reference

18 August 2017

24

Stéphane Blanche

1 reference

18 August 2017

11

Anne-Marie McNicol

1 reference

18 August 2017

17

Annick Lim

1 reference

18 August 2017

H Bobby Gaspar

2

1 reference

18 August 2017

Sabine Charrier

5

1 reference

18 August 2017

Karen Buckland

6

1 reference

18 August 2017

Nourredine Himoudi

9

1 reference

18 August 2017

Kimberly Gilmour

10

1 reference

18 August 2017

Havinder Hara

12

1 reference

18 August 2017

Jinhua Xu-Bayford

13

1 reference

18 August 2017

Christine Rivat

14

1 reference

18 August 2017

Jean-Marc Treluyer

18

1 reference

18 August 2017

Francois Lefrère

20

1 reference

18 August 2017

Jeremy Magalon

21

1 reference

18 August 2017

Isabelle Pengue-Koyi

22

1 reference

18 August 2017

Géraldine Honnet

23

1 reference

18 August 2017

Eric A Sherman

25

1 reference

18 August 2017

Frances Male

26

1 reference

18 August 2017

Charles Berry

27

1 reference

18 August 2017

Nirav Malani

28

1 reference

18 August 2017

Frederic D Bushman

29

1 reference

18 August 2017

Anne Galy

32

1 reference

18 August 2017

language of work or name

English

0 references

publication date

1 April 2015

1 reference

The Journal of the American Medical Association

18 August 2017

1 reference

18 August 2017

313

1 reference

18 August 2017

15

1 reference

18 August 2017

1550-1563

1 reference

B-cell subpopulations in children: National reference values

18 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

A modified γ-retrovirus vector for X-linked severe combined immunodeficiency

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Comparing DNA integration site clusters with scan statistics.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Estimating abundances of retroviral insertion sites from DNA fragment length data.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Quantification of lentiviral vector copy numbers in individual hematopoietic colony-forming cells shows vector dose-dependent effects on the frequency and level of transduction.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Clinical spectrum, pathophysiology and treatment of the Wiskott–Aldrich syndrome

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Large-scale manufacture and characterization of a lentiviral vector produced for clinical ex vivo gene therapy application

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Efficacy of gene therapy for X-linked severe combined immunodeficiency

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: advances in biology and future directions for treatment

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

WASP: a key immunological multitasker

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

WASP confers selective advantage for specific hematopoietic cell populations and serves a unique role in marginal zone B-cell homeostasis and function

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

HIV integration site selection: analysis by massively parallel pyrosequencing reveals association with epigenetic modifications

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

The Wiskott-Aldrich syndrome protein is required for the function of CD4(+)CD25(+)Foxp3(+) regulatory T cells

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Selection of target sites for mobile DNA integration in the human genome

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Wiskott-Aldrich syndrome protein and the cytoskeletal dynamics of dendritic cells

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Clinical course of patients with WASP gene mutations

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Configuration of human dendritic cell cytoskeleton by Rho GTPases, the WAS protein, and differentiation

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Actin cytoskeletal function is spared, but apoptosis is increased, in WAS patient hematopoietic cells.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Antigen receptor-induced activation and cytoskeletal rearrangement are impaired in Wiskott-Aldrich syndrome protein-deficient lymphocytes

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

T cells of patients with the Wiskott-Aldrich syndrome have a restricted defect in proliferative responses.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

The Wiskott-Aldrich syndrome and X-linked congenital thrombocytopenia are caused by mutations of the same gene

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better?

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Results and long-term outcome in 39 patients with Wiskott-Aldrich syndrome transplanted from HLA-matched and -mismatched donors

3 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

WASP regulates suppressor activity of human and murine CD4(+)CD25(+)FOXP3(+) natural regulatory T cells

3 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Evidence for adequate thymic function but impaired naive T-cell survival following allogeneic hematopoietic stem cell transplantation in the absence of chronic graft-versus-host disease

3 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Mixed chimera status of 12 patients with Wiskott-Aldrich syndrome (WAS) after hematopoietic stem cell transplantation: evaluation by flow cytometric analysis of intracellular WAS protein expression

3 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Wiskott-Aldrich syndrome protein-deficient mice reveal a role for WASP in T but not B cell activation

3 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation

3 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4942841

Outcome in patients with Wiskott-Aldrich syndrome following stem cell transplantation: an analysis of 57 patients in Japan

4 December 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25898053

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Combination of MHC-peptide multimer-based T cell sorting with the Immunoscope permits sensitive ex vivo quantitation and follow-up of human CD8+ T cell immune responses

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25898053

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1001/JAMA.2015.3253

1 reference

18 August 2017

1 reference

18 August 2017

PubMed publication ID

25898053

1 reference