Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome.
Adolescent
Adult
Animals
Antibodies, Neutralizing
/ biosynthesis
Antibodies, Viral
/ biosynthesis
Bilirubin
/ immunology
Capsid
/ immunology
Child
Child, Preschool
Crigler-Najjar Syndrome
/ genetics
Dependovirus
/ genetics
Excitatory Amino Acid Antagonists
/ therapeutic use
Female
Gene Expression
Genetic Therapy
/ methods
Glucuronosyltransferase
/ deficiency
HEK293 Cells
Humans
Immunity, Innate
Immunization, Passive
Liver
/ immunology
Male
Mice
Mice, Inbred C57BL
Phenobarbital
/ therapeutic use
Phototherapy
/ methods
Plasmids
/ chemistry
Transfection
AAV gene therapy
Crigler–Najjar syndrome
UGT1A1
anti-AAV neutralizing antibodies
pre-existing immunity
unconjugated hyperbilirubinemia
Journal
Human gene therapy
ISSN: 1557-7422
Titre abrégé: Hum Gene Ther
Pays: United States
ID NLM: 9008950
Informations de publication
Date de publication:
10 2019
10 2019
Historique:
pubmed:
11
9
2019
medline:
28
3
2020
entrez:
11
9
2019
Statut:
ppublish
Résumé
Adeno-associated virus (AAV) vector-mediated gene therapy is currently evaluated as a potential treatment for Crigler-Najjar syndrome (CN) (NCT03466463). Pre-existing immunity to AAV is known to hinder gene transfer efficacy, restricting enrollment of seropositive subjects in ongoing clinical trials. We assessed the prevalence of anti-AAV serotype 8 (AAV8) neutralizing antibodies (NAbs) in subjects affected by CN and investigated the impact of low NAb titers (<1:5) on liver gene transfer efficacy in an
Identifiants
pubmed: 31502485
doi: 10.1089/hum.2019.143
pmc: PMC6763963
doi:
Substances chimiques
Antibodies, Neutralizing
0
Antibodies, Viral
0
Excitatory Amino Acid Antagonists
0
UGT1A1 enzyme
EC 2.4.1.-
Glucuronosyltransferase
EC 2.4.1.17
Bilirubin
RFM9X3LJ49
Phenobarbital
YQE403BP4D
Types de publication
Journal Article
Multicenter Study
Observational Study
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
1297-1305Références
Nat Med. 2007 Apr;13(4):419-22
pubmed: 17369837
N Engl J Med. 2008 Feb 28;358(9):920-8
pubmed: 18305267
Gene Ther. 2014 Feb;21(2):168-74
pubmed: 24285217
Blood. 2013 Jul 4;122(1):23-36
pubmed: 23596044
J Immunol. 2012 Jun 15;188(12):6418-24
pubmed: 22593612
Mol Ther. 2010 Nov;18(11):1983-94
pubmed: 20736932
Gene Ther. 2012 Mar;19(3):288-94
pubmed: 21697954
Curr Gene Ther. 2010 Dec;10(6):423-36
pubmed: 21054248
Mol Ther Methods Clin Dev. 2018 Dec 31;12:157-174
pubmed: 30705921
Pediatrics. 1952 Aug;10(2):169-80
pubmed: 12983120
Cell Immunol. 2019 Aug;342:103780
pubmed: 29571923
Mol Ther. 2011 Nov;19(11):2084-91
pubmed: 21629225
N Engl J Med. 2017 Dec 7;377(23):2215-2227
pubmed: 29211678
Blood. 2006 Nov 15;108(10):3321-8
pubmed: 16868252
Sci Transl Med. 2013 Jul 17;5(194):194ra92
pubmed: 23863832
J Med Virol. 2009 Jan;81(1):65-74
pubmed: 19031458
Hum Gene Ther. 2006 Apr;17(4):440-7
pubmed: 16610931
J Infect Dis. 2009 Feb 1;199(3):381-90
pubmed: 19133809
Proc Natl Acad Sci U S A. 2003 May 13;100(10):6081-6
pubmed: 12716974
Mol Ther. 2014 Feb;22(2):338-347
pubmed: 24196577
Hum Gene Ther. 2010 Jun;21(6):704-12
pubmed: 20095819
Hum Gene Ther. 2017 Nov;28(11):1061-1074
pubmed: 28835127
Hum Gene Ther Methods. 2013 Apr;24(2):59-67
pubmed: 23442094
Mol Ther. 2006 Jun;13(6):1085-92
pubmed: 16581301
Nat Commun. 2018 Oct 5;9(1):4098
pubmed: 30291246
N Engl J Med. 2017 Nov 2;377(18):1713-1722
pubmed: 29091557
Mol Ther. 2011 May;19(5):876-85
pubmed: 21245849
Hum Gene Ther Methods. 2015 Apr;26(2):45-53
pubmed: 25819687
Eur J Pediatr. 2006 May;165(5):306-19
pubmed: 16435131
Hepatology. 1996 Aug;24(2):311-5
pubmed: 8690398
J Biol Chem. 1994 Jul 8;269(27):17960-4
pubmed: 8027054
J Virol Methods. 2014 Feb;196:163-73
pubmed: 24239634
Blood. 2018 Mar 1;131(9):1022-1031
pubmed: 29246900
N Engl J Med. 2008 May 22;358(21):2240-8
pubmed: 18441370
Hum Gene Ther. 2011 Nov;22(11):1389-401
pubmed: 21476868
N Engl J Med. 2013 Nov 21;369(21):2021-30
pubmed: 24256380
N Engl J Med. 2014 Nov 20;371(21):1994-2004
pubmed: 25409372
N Engl J Med. 2008 May 22;358(21):2231-9
pubmed: 18441371
Mol Ther Methods Clin Dev. 2016 Jul 20;3:16049
pubmed: 27722180
Hum Gene Ther. 2015 Mar;26(3):145-52
pubmed: 25654180
N Engl J Med. 2017 Dec 28;377(26):2519-2530
pubmed: 29224506
Blood. 2006 Mar 1;107(5):1810-7
pubmed: 16249376
Mol Ther Methods Clin Dev. 2018 Feb 13;9:119-129
pubmed: 29766022
Nat Med. 2006 Mar;12(3):342-7
pubmed: 16474400
Hum Gene Ther. 2014 Sep;25(9):844-55
pubmed: 25072305