(Q35568541)

8 August 2017

title

Retroviral gene therapy for X-linked chronic granulomatous disease: results from phase I/II trial (English)

1 reference

X-linked chronic granulomatous disease

8 August 2017

main subject

phase I clinical trial

0 references

gene therapy

0 references

phase II clinical trial

0 references

0 references

12

Christof Von Kalle

1 reference

8 August 2017

13

Manfred Schmidt

1 reference

8 August 2017

15

Hyo Seop Ahn

1 reference

8 August 2017

Hyoung Jin Kang

1

1 reference

8 August 2017

Cynthia C Bartholomae

2

1 reference

8 August 2017

Anna Paruzynski

3

1 reference

8 August 2017

Anne Arens

4

1 reference

8 August 2017

Sujeong Kim

5

1 reference

8 August 2017

Seung Shin Yu

6

1 reference

8 August 2017

Youngtae Hong

7

1 reference

8 August 2017

Chang-Wan Joo

8

1 reference

8 August 2017

Nam-Kyung Yoon

9

1 reference

8 August 2017

Jung-Woo Rhim

10

1 reference

8 August 2017

Joong Gon Kim

11

1 reference

8 August 2017

Sunyoung Kim

14

1 reference

8 August 2017

language of work or name

1 reference

30 August 2011

1 reference

8 August 2017

1 reference

8 August 2017

volume

19

1 reference

8 August 2017

issue

11

1 reference

8 August 2017

page(s)

2092-2101

1 reference

Chronic granulomatous disease: recent advances in pathophysiology and treatment

8 August 2017

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Gene therapy of chronic granulomatous disease: the engraftment dilemma

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

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

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

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

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

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

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

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

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Retrospective analysis of peripheral blood stem cell transplantation for the treatment of high-risk neuroblastoma

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

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

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Insertional mutagenesis identifies genes that promote the immortalization of primary bone marrow progenitor cells

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Autologous peripheral blood stem cell transplantation with BCVAC conditioning in childhood acute myeloid leukemia

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Chronic granulomatous disease.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Construction of a retroviral vector production system with the minimum possibility of a homologous recombination

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

The superoxide-generating NADPH oxidase: structural aspects and activation mechanism

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Once-daily intravenous busulfan given with fludarabine as conditioning for allogeneic stem cell transplantation: study of pharmacokinetics and early clinical outcomes

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Chronic granulomatous disease. Report on a national registry of 368 patients

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

High efficiency retroviral vectors that contain no viral coding sequences

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

A transient three-plasmid expression system for the production of high titer retroviral vectors

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Molecular basis of chronic granulomatous disease

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Advances in treatment for chronic granulomatous disease

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

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

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Early engraftment kinetics of two units cord blood transplantation.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

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

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Once-daily intravenous busulfan and fludarabine: clinical and pharmacokinetic results of a myeloablative, reduced-toxicity conditioning regimen for allogeneic stem cell transplantation in AML and MDS.

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Conditioning with fludarabine and targeted busulfan for transplantation of allogeneic hematopoietic stem cells

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

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

26 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3222528

Gene therapy for chronic granulomatous disease

26 September 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21878903

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Monoclonal antibody 7D5 raised to cytochrome b558 of human neutrophils: immunocytochemical detection of the antigen in peripheral phagocytes of normal subjects, patients with chronic granulomatous disease, and their carrier mothers

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21878903

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Genetically corrected autologous stem cells engraft, but host immune responses limit their utility in canine alpha-L-iduronidase deficiency

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21878903

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/MT.2011.166

1 reference

8 August 2017

1 reference

8 August 2017

1 reference