Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy (Q45872766)

29 June 2018

title

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy (English)

1 reference

29 June 2018

0 references

Satiro De Oliveira

5

0 references

Adrian Thrasher

object named as

Adrian J Thrasher

6

0 references

Paul Gissen

object named as

Paul Gissen

16

0 references

author name string

Florian Eichler

1

1 reference

29 June 2018

Christine Duncan

2

1 reference

29 June 2018

Patricia L Musolino

3

1 reference

29 June 2018

Paul J Orchard

4

1 reference

29 June 2018

Myriam Armant

7

1 reference

29 June 2018

Colleen Dansereau

8

1 reference

29 June 2018

Troy C Lund

9

1 reference

29 June 2018

Weston P Miller

10

1 reference

29 June 2018

Gerald V Raymond

11

1 reference

29 June 2018

Raman Sankar

12

1 reference

29 June 2018

Ami J Shah

13

1 reference

29 June 2018

Caroline Sevin

14

1 reference

29 June 2018

H Bobby Gaspar

15

1 reference

29 June 2018

Hernan Amartino

17

1 reference

29 June 2018

Drago Bratkovic

18

1 reference

29 June 2018

Nicholas J C Smith

19

1 reference

29 June 2018

Asif M Paker

20

1 reference

29 June 2018

Esther Shamir

21

1 reference

29 June 2018

Tara O'Meara

22

1 reference

29 June 2018

David Davidson

23

1 reference

29 June 2018

Patrick Aubourg

24

1 reference

29 June 2018

David A Williams

25

1 reference

29 June 2018

language of work or name

English

0 references

publication date

4 October 2017

1 reference

29 June 2018

The New England Journal of Medicine

published in

1 reference

29 June 2018

volume

377

1 reference

29 June 2018

issue

17

1 reference

29 June 2018

page(s)

1630-1638

1 reference

29 June 2018

Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and Hurler syndrome.

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

Gene therapy returns to centre stage

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

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

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

Hematopoietic cell transplantation does not prevent myelopathy in X-linked adrenoleukodystrophy: a retrospective study

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

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

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

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

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

X-linked adrenoleukodystrophy (X-ALD): clinical presentation and guidelines for diagnosis, follow-up and management.

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

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

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

Ex vivo gene transfer and correction for cell-based therapies

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

Early diagnosis of cerebral X-linked adrenoleukodystrophy in boys with Addison's disease improves survival and neurological outcomes

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

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

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

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

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

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

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

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

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

X-Linked adrenoleukodystrophy: overview and prognosis as a function of age and brain magnetic resonance imaging abnormality. A study involving 372 patients

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

Plasma very long chain fatty acids in 3,000 peroxisome disease patients and 29,000 controls

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

Adrenoleukodystrophy: phenotype, genetics, pathogenesis and therapy

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

Adrenoleukodystrophy: a scoring method for brain MR observations

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

The inflammatory myelinopathy of adreno-leukodystrophy: cells, effector molecules, and pathogenetic implications

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

Outcomes of haematopoietic stem cell transplantation for inherited metabolic disorders: a report from the Australian and New Zealand Children's Haematology Oncology Group and the Australasian Bone Marrow Transplant Recipient Registry

30 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

Is microglial apoptosis an early pathogenic change in cerebral X-linked adrenoleukodystrophy?

15 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

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

15 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

Outcomes of unrelated umbilical cord blood transplantation for X-linked adrenoleukodystrophy.

15 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999.

15 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

Analysis of MRI patterns aids prediction of progression in X-linked adrenoleukodystrophy

15 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5708849

Haematopoietic stem cell transplantation in 12 patients with cerebral X-linked adrenoleukodystrophy

15 August 2018

1 reference

12 December 2020

Survival analysis of haematopoietic cell transplantation for childhood cerebral X-linked adrenoleukodystrophy: a comparison study

1 reference

12 December 2020

Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report

1 reference

12 December 2020

Long-term effect of bone-marrow transplantation for childhood-onset cerebral X-linked adrenoleukodystrophy

1 reference

12 December 2020

Identifiers

DOI

10.1056/NEJMOA1700554

1 reference

29 June 2018

release_elslpaq2nzbgxnssoftltovfii

Fatcat ID

1 reference

Fatcat

https://api.fatcat.wiki/v0/release/elslpaq2nzbgxnssoftltovfii

24 November 2022

mapped directly with Wikidata item

PMCID

5708849

1 reference

29 June 2018

PubMed ID

28976817

1 reference

29 June 2018