Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients (Q36812729)

16 August 2017

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

1 reference

16 August 2017

0 references

25

Christof von Kalle

0 references

Martijn Brugman

Martijn H Brugman

7

0 references

Kimberly Gilmour

Kimberly C Gilmour

11

0 references

Marc Mansour

Marc R Mansour

2

0 references

Michael Hubank

Michael Hubank

5

0 references

Adrian Thrasher

Adrian J Thrasher

27

0 references

Manfred Schmidt

24

Manfred Schmidt

1 reference

16 August 2017

17

David C Linch

1 reference

16 August 2017

16

Rosemary E Gale

1 reference

16 August 2017

26

H Bobby Gaspar

1 reference

16 August 2017

18

Jinhua Bayford

1 reference

16 August 2017

Cynthia C Bartholomae

4

Cynthia Bartholomae

1 reference

16 August 2017

Kerstin Schwarzwaelder

3

Kerstin Schwarzwaelder

1 reference

16 August 2017

14

Kathryn L Parsley

1 reference

16 August 2017

8

Karin Pike-Overzet

1 reference

16 August 2017

15

Frank J T Staal

1 reference

16 August 2017

10

Dick de Ridder

1 reference

16 August 2017

13

Susannah I Thornhill

1 reference

16 August 2017

Steven J Howe

1

1 reference

16 August 2017

Helena Kempski

6

1 reference

16 August 2017

Stephen J Chatters

9

1 reference

16 August 2017

Stuart Adams

12

1 reference

16 August 2017

Lucie Brown

19

1 reference

16 August 2017

Michelle Quaye

20

1 reference

16 August 2017

Christine Kinnon

21

1 reference

16 August 2017

Philip Ancliff

22

1 reference

16 August 2017

David K Webb

23

1 reference

16 August 2017

language of work or name

English

0 references

publication date

1 September 2008

1 reference

Journal of Clinical Investigation

16 August 2017

1 reference

16 August 2017

118

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16 August 2017

3143-3150

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16 August 2017

9

1 reference

NKG2D-deficient mice are defective in tumor surveillance in models of spontaneous malignancy

16 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Self-inactivating gammaretroviral vectors for gene therapy of 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=2496964

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 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=2496964

Hot spots of retroviral integration in human CD34+ 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=2496964

Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in 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=2496964

Exploration, normalization, and genotype calls of high-density oligonucleotide SNP array 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=2496964

Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Unique risk factors for insertional mutagenesis in a mouse model of XSCID 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=2496964

Cell-culture assays reveal the importance of retroviral vector design for insertional 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=2496964

Leukemia-associated mutations within the NOTCH1 heterodimerization domain fall into at least two distinct mechanistic classes

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Notch 1 activation in the molecular pathogenesis of T-cell acute lymphoblastic leukaemia

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

The role of LMO2 in development and in T cell leukemia after chromosomal translocation or retroviral insertion

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Activating Notch1 mutations in mouse models of T-ALL.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Interleukin-7 in T-cell acute lymphoblastic leukemia: an extrinsic factor supporting leukemogenesis?

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Prospective gene expression analysis accurately subtypes acute leukaemia in children and establishes a commonality between hyperdiploidy and t(12;21) in acute lymphoblastic leukaemia

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Activating mutations of NOTCH1 in human T cell acute lymphoblastic leukemia

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Bioconductor: open software development for computational biology and bioinformatics

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Transcription start regions in the human genome are favored targets for MLV integration

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

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=2496964

V(D)J-mediated translocations in lymphoid neoplasms: a functional assessment of genomic instability by cryptic sites.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

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=2496964

Cloning of the gamma chain of the human IL-2 receptor

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Interleukin-2 receptor gamma chain mutation results in X-linked severe combined immunodeficiency in humans

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

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

27 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

NOTCH1 mutation can be an early, prenatal genetic event in T-ALL.

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Ectopic retroviral expression of LMO2, but not IL2Rgamma, blocks human T-cell development from CD34+ cells: implications for leukemogenesis in gene therapy.

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

New insights on human T cell development by quantitative T cell receptor gene rearrangement studies and gene expression profiling.

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Gene therapy insertional mutagenesis insights

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

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

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

The oncogenic LIM protein Rbtn2 causes thymic developmental aberrations that precede malignancy in transgenic mice.

5 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2496964

T-cell re-population in HIV-infected children on highly active anti-retroviral therapy (HAART)

5 September 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/18688286

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Notch-1 mutations are secondary events in some patients with T-cell acute lymphoblastic leukemia

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/18688286

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1172/JCI35798

1 reference

16 August 2017

1 reference

16 August 2017

1 reference