Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47
biodistribution
chronic granulomatous disease
genotoxicity
lentiviral gene therapy
Journal
Human gene therapy
ISSN: 1557-7422
Titre abrégé: Hum Gene Ther
Pays: United States
ID NLM: 9008950
Informations de publication
Date de publication:
09 2021
09 2021
Historique:
pubmed:
21
3
2021
medline:
1
2
2022
entrez:
20
3
2021
Statut:
ppublish
Résumé
Chronic granulomatous disease (CGD) is an inherited blood disorder of phagocytic cells that renders patients susceptible to infections and inflammation. A recent clinical trial of lentiviral gene therapy for the most frequent form of CGD, X-linked, has demonstrated stable correction over time, with no adverse events related to the gene therapy procedure. We have recently developed a parallel lentiviral vector for p47
Identifiants
pubmed: 33740872
doi: 10.1089/hum.2020.276
pmc: PMC8575060
doi:
Substances chimiques
NADPH Oxidases
EC 1.6.3.-
Types de publication
Journal Article
Research Support, N.I.H., Intramural
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
949-958Subventions
Organisme : Wellcome Trust
ID : 217112/Z/19/Z
Pays : United Kingdom
Organisme : Intramural NIH HHS
ID : ZIA AI000989
Pays : United States
Organisme : Intramural NIH HHS
ID : ZIA AI000644
Pays : United States
Références
Cell Stem Cell. 2018 Dec 6;23(6):820-832.e9
pubmed: 30416070
Mol Ther. 2009 Nov;17(11):1919-28
pubmed: 19672245
Nat Med. 2006 Apr;12(4):401-9
pubmed: 16582916
J Pediatric Infect Dis Soc. 2018 May 9;7(suppl_1):S2-S5
pubmed: 29746675
J Allergy Clin Immunol. 2013 Nov;132(5):1156-1163.e5
pubmed: 23910690
J Clin Immunol. 2011 Aug;31(4):699-709
pubmed: 21544592
Gene Ther. 2020 Sep;27(9):459-469
pubmed: 32533104
Scand J Immunol. 2019 Feb;89(2):e12737
pubmed: 30506560
Hum Gene Ther Clin Dev. 2017 Mar;28(1):17-27
pubmed: 28319446
N Engl J Med. 2010 Dec 30;363(27):2600-10
pubmed: 21190454
J Immunol. 2012 May 15;188(10):5003-11
pubmed: 22491245
J Am Assoc Lab Anim Sci. 2010 Jul;49(4):480-6
pubmed: 20819397
Mol Ther. 2011 Jan;19(1):122-32
pubmed: 20978475
Mol Ther. 2021 Jan 6;29(1):86-102
pubmed: 33010230
Nat Med. 2010 Feb;16(2):198-204
pubmed: 20098431
Mol Ther Methods Clin Dev. 2019 Apr 04;13:390-398
pubmed: 31024981
Hum Gene Ther. 2016 Sep;27(9):668-78
pubmed: 27056660
Front Pediatr. 2020 Jun 26;8:327
pubmed: 32676488
Blood. 2006 Oct 15;108(8):2545-53
pubmed: 16825499
Scand J Immunol. 2019 Jul;90(1):e12767
pubmed: 30963593
J Allergy Clin Immunol. 2018 Jan;141(1):365-371
pubmed: 28528201
Am J Pathol. 2009 Jan;174(1):153-63
pubmed: 19095958
Nat Med. 2020 Feb;26(2):200-206
pubmed: 31988463
Mol Ther Methods Clin Dev. 2019 Jun 07;14:134-147
pubmed: 31338385
J Gene Med. 2012 Aug;14(8):549-60
pubmed: 22887595
Adv Ther. 2017 Dec;34(12):2543-2557
pubmed: 29168144
Blood. 2019 Jun 6;133(23):2546-2549
pubmed: 30952673
Hum Gene Ther Clin Dev. 2018 Jun;29(2):69-79
pubmed: 29664709