Lentiviral gene therapy for X-linked chronic granulomatous disease.
Adolescent
Antigens, CD34
/ genetics
Child
Child, Preschool
Chromosomes, Human, X
Comorbidity
Gene Silencing
Genes, Regulator
Genetic Therapy
/ methods
Genetic Vectors
Granulomatous Disease, Chronic
/ genetics
Hematopoietic Stem Cells
/ cytology
Humans
Lentivirus
/ genetics
Male
NADPH Oxidases
/ genetics
Neutrophils
/ metabolism
Patient Safety
Promoter Regions, Genetic
Transplantation Conditioning
Treatment Outcome
United Kingdom
United States
Young Adult
Journal
Nature medicine
ISSN: 1546-170X
Titre abrégé: Nat Med
Pays: United States
ID NLM: 9502015
Informations de publication
Date de publication:
02 2020
02 2020
Historique:
received:
18
07
2019
accepted:
10
12
2019
pubmed:
29
1
2020
medline:
22
4
2020
entrez:
29
1
2020
Statut:
ppublish
Résumé
Chronic granulomatous disease (CGD) is a rare inherited disorder of phagocytic cells
Identifiants
pubmed: 31988463
doi: 10.1038/s41591-019-0735-5
pii: 10.1038/s41591-019-0735-5
pmc: PMC7115833
mid: EMS86478
doi:
Substances chimiques
Antigens, CD34
0
NADPH Oxidases
EC 1.6.3.-
Types de publication
Clinical Trial, Phase I
Clinical Trial, Phase II
Journal Article
Multicenter Study
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
200-206Subventions
Organisme : Wellcome Trust
ID : 104807
Pays : United Kingdom
Organisme : NCI NIH HHS
ID : K08 CA201591
Pays : United States
Investigateurs
Karen F Buckland
(KF)
Christopher A Bauser
(CA)
Geraldine Honnet
(G)
Manuel Grez
(M)
H Bobby Gaspar
(HB)
Anne Galy
(A)
Adrian J Thrasher
(AJ)
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