Targeting a Pre-existing Anti-transgene T Cell Response for Effective Gene Therapy of MPS-I in the Mouse Model of the Disease.
Animals
Antibodies, Monoclonal
/ pharmacology
CD8-Positive T-Lymphocytes
/ immunology
Cells, Cultured
Disease Models, Animal
Enzyme Replacement Therapy
/ adverse effects
Gene Knockout Techniques
Genetic Therapy
/ methods
Genetic Vectors
Hematopoietic Stem Cell Transplantation
/ methods
Humans
Iduronidase
/ genetics
Immunity, Cellular
/ drug effects
Immunization
/ methods
Immunoglobulin G
/ blood
Mice
Mice, Inbred C57BL
Mice, Knockout
Mucopolysaccharidosis I
/ therapy
Spleen
/ pathology
Transgenes
/ immunology
ERT
HSC
MPS-I
depleting agents
fludarabine
gene therapy
immune response
lentiviral vector
pre-existing immunity
Journal
Molecular therapy : the journal of the American Society of Gene Therapy
ISSN: 1525-0024
Titre abrégé: Mol Ther
Pays: United States
ID NLM: 100890581
Informations de publication
Date de publication:
03 07 2019
03 07 2019
Historique:
received:
01
02
2019
revised:
12
04
2019
accepted:
14
04
2019
pubmed:
8
5
2019
medline:
27
2
2020
entrez:
8
5
2019
Statut:
ppublish
Résumé
Mucopolysaccharidosis type I (MPS-I) is a severe genetic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) enzyme. Ex vivo hematopoietic stem cell (HSC) gene therapy is a promising therapeutic approach for MPS-I, as demonstrated by preclinical studies performed in naive MPS-I mice. However, after enzyme replacement therapy (ERT), several MPS-I patients develop anti-IDUA immunity that may jeopardize ex vivo gene therapy efficacy. Here we treat MPS-I mice with an artificial immunization protocol to mimic the ERT effect in patients, and we demonstrate that IDUA-corrected HSC engraftment is impaired in pre-immunized animals by IDUA-specific CD8
Identifiants
pubmed: 31060789
pii: S1525-0016(19)30180-7
doi: 10.1016/j.ymthe.2019.04.014
pmc: PMC6612662
pii:
doi:
Substances chimiques
Antibodies, Monoclonal
0
Immunoglobulin G
0
Iduronidase
EC 3.2.1.76
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
1215-1227Informations de copyright
Copyright © 2019 The Authors. Published by Elsevier Inc. All rights reserved.
Références
J Pharm Sci. 2009 Dec;98(12):4480-4
pubmed: 19499565
Mol Genet Metab. 2013 May;109(1):33-40
pubmed: 23562162
Proc Natl Acad Sci U S A. 2003 Feb 18;100(4):1902-7
pubmed: 12576554
Mol Genet Metab. 2009 Jan;96(1):13-9
pubmed: 19038563
Anticancer Res. 1998 Jul-Aug;18(4A):2345-51
pubmed: 9703877
Lancet. 2016 Jul 30;388(10043):476-87
pubmed: 27289174
Pediatrics. 2009 Jan;123(1):229-40
pubmed: 19117887
Blood. 2015 Mar 26;125(13):2164-72
pubmed: 25624320
PLoS One. 2013 Jun 25;8(6):e67052
pubmed: 23825616
Adv Drug Deliv Rev. 2017 Sep 1;118:109-134
pubmed: 28502768
J Exp Med. 2011 Nov 21;208(12):2357-66
pubmed: 22065672
J Pediatr. 2004 May;144(5):581-8
pubmed: 15126990
Muscle Nerve. 2011 Nov;44(5):720-6
pubmed: 21952916
Bone Marrow Transplant. 2008 Mar;41(6):531-5
pubmed: 18037941
Biol Blood Marrow Transplant. 2014 Mar;20(3):345-53
pubmed: 24315842
Biol Blood Marrow Transplant. 2005 Jun;11(6):465-71
pubmed: 15931635
Nature. 2015 Oct 15;526(7573):351-60
pubmed: 26469046
N Engl J Med. 2006 Apr 27;354(17):1813-26
pubmed: 16641398
Mol Genet Metab. 2016 Apr;117(4):419-26
pubmed: 26920513
Nat Med. 2001 Jan;7(1):33-40
pubmed: 11135613
Transplantation. 2005 Nov 15;80(9):1145-52
pubmed: 16314778
Gene Ther. 2008 Jun;15(11):808-16
pubmed: 18385765
Annu Rev Immunol. 1994;12:991-1045
pubmed: 8011301
Immunol Rev. 1997 Apr;156:199-209
pubmed: 9176709
Mol Genet Metab. 2016 Feb;117(2):66-83
pubmed: 26597321
Mol Genet Metab. 2010 Jan;99(1):26-33
pubmed: 19775921
Biol Blood Marrow Transplant. 2015 Jun;21(6):1106-9
pubmed: 25708213
Clin Immunol. 2013 Dec;149(3):534-55
pubmed: 24263283
Mol Genet Metab. 2003 Sep-Oct;80(1-2):148-58
pubmed: 14567964
Nat Biotechnol. 2005 Jan;23(1):108-16
pubmed: 15619618
Blood. 2013 Jul 4;122(1):23-36
pubmed: 23596044
Science. 2013 Aug 23;341(6148):1233151
pubmed: 23845947
J Immunol. 2002 Oct 1;169(7):3760-70
pubmed: 12244170
Hum Mutat. 1995;6(4):288-302
pubmed: 8680403
Mol Genet Metab. 2016 Mar;117(3):373-7
pubmed: 26832957
Springer Semin Immunopathol. 2005 Jan;26(3):247-55
pubmed: 15609002
Mol Ther. 2007 May;15(5):889-902
pubmed: 17311010
Scand J Immunol. 2007 Apr;65(4):311-9
pubmed: 17386021
Mol Ther. 2006 Jul;14(1):5-13
pubmed: 16698321
Front Immunol. 2014 Apr 01;5:140
pubmed: 24744762
Transl Res. 2013 Apr;161(4):230-40
pubmed: 23360745
Science. 2013 Aug 23;341(6148):1233158
pubmed: 23845948
Mol Ther. 2012 Oct;20(10):1924-31
pubmed: 22871665
J Immunol. 2011 Oct 15;187(8):4100-8
pubmed: 21930972
Mol Genet Metab. 2007 Feb;90(2):171-80
pubmed: 17011223
Trends Mol Med. 2003 Oct;9(10):450-3
pubmed: 14557058
Pharmacotherapy. 1998 Sep-Oct;18(5):988-1000
pubmed: 9758310
Mol Ther Methods Clin Dev. 2017 Oct 05;8:42-51
pubmed: 29159202
Trends Pharmacol Sci. 2010 Feb;31(2):53-9
pubmed: 19963283
Gene Ther. 2010 Mar;17(3):295-304
pubmed: 19907498
Transplantation. 1992 Nov;54(5):844-51
pubmed: 1440852
Blood. 2010 Dec 9;116(24):5130-9
pubmed: 20847202
Adv Drug Deliv Rev. 1998 Jul 6;32(3):173-186
pubmed: 10837643
Immunology. 1962 Jan;5:161-8
pubmed: 13887797
Biomed Res Int. 2016;2016:3071214
pubmed: 27843940
J Thromb Haemost. 2012 Aug;10(8):1570-80
pubmed: 22632092
Nat Rev Genet. 2011 May;12(5):316-28
pubmed: 21468099
Springer Semin Immunopathol. 2005 Jan;26(3):231-46
pubmed: 15609001
Lancet. 2003 May 10;361(9369):1608-13
pubmed: 12747881
Blood. 2007 Sep 15;110(6):1788-96
pubmed: 17495135
Mol Genet Metab. 2003 Aug;79(4):233-44
pubmed: 12948739