Enhanced Factor IX Activity following Administration of AAV5-R338L "Padua" Factor IX versus AAV5 WT Human Factor IX in NHPs.
AAV
AAV5
AMT-060
AMT-061
adeno-associated virus
factor IX
gene therapy
hemophilia B
non-human primate
Journal
Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857
Informations de publication
Date de publication:
13 Dec 2019
13 Dec 2019
Historique:
received:
17
05
2019
accepted:
19
09
2019
entrez:
12
11
2019
pubmed:
12
11
2019
medline:
12
11
2019
Statut:
epublish
Résumé
Gene therapy for severe hemophilia B is advancing and offers sustained disease amelioration with a single treatment. We have reported the efficacy and safety of AMT-060, an investigational gene therapy comprising an adeno-associated virus serotype 5 capsid encapsidating the codon-optimized wild-type human factor IX (WT h
Identifiants
pubmed: 31709273
doi: 10.1016/j.omtm.2019.09.005
pii: S2329-0501(19)30103-2
pmc: PMC6834974
doi:
Types de publication
Journal Article
Langues
eng
Pagination
221-231Informations de copyright
© 2019 The Authors.
Références
Blood. 2009 Oct 1;114(14):2878-83
pubmed: 19617576
N Engl J Med. 2007 Aug 9;357(6):603-5
pubmed: 17687136
Blood Adv. 2018 Mar 13;2(5):505-508
pubmed: 29500218
Biometrics. 1971 Mar;27(1):103-17
pubmed: 5547548
Mol Ther Methods Clin Dev. 2019 May 28;14:27-36
pubmed: 31276009
N Engl J Med. 2001 Jun 7;344(23):1773-9
pubmed: 11396445
Hum Gene Ther. 2006 Apr;17(4):440-7
pubmed: 16610931
Haemophilia. 2013 Jan;19(1):e1-47
pubmed: 22776238
Biometrics. 1972 Jun;28(2):519-31
pubmed: 5037867
N Engl J Med. 2007 Aug 9;357(6):535-44
pubmed: 17687129
Lab Anim Res. 2016 Jun;32(2):79-86
pubmed: 27382375
Hum Gene Ther. 2010 Jun;21(6):704-12
pubmed: 20095819
Prog Mol Biol Transl Sci. 2012;105:151-209
pubmed: 22137432
Clin Immunol. 2009 May;131(2):189-201
pubmed: 19269256
Blood. 1998 Jul 1;92(1):168-74
pubmed: 9639513
Proc Soc Exp Biol Med. 1952 Apr;79(4):692-4
pubmed: 14920537
N Engl J Med. 2009 Oct 22;361(17):1671-5
pubmed: 19846852
Hum Gene Ther Clin Dev. 2018 Jun;29(2):80-89
pubmed: 29624465
Mol Ther. 2017 Aug 2;25(8):1831-1842
pubmed: 28596114
Clin Immunol. 2007 Jul;124(1):26-32
pubmed: 17490912
Haemophilia. 2011 Jan;17(1):2-10
pubmed: 20731726
Blood Adv. 2017 Dec 08;1(26):2591-2599
pubmed: 29296912
Mol Ther. 2011 May;19(5):876-85
pubmed: 21245849
Proc Natl Acad Sci U S A. 1997 Oct 14;94(21):11563-6
pubmed: 9326649
Transfusion. 2015 Jul;55(7):1787-97
pubmed: 25652955
Blood. 2005 Apr 15;105(8):3079-86
pubmed: 15637142
Blood. 2013 Apr 25;121(17):3335-44
pubmed: 23426947
Blood. 2015 Mar 5;125(10):1553-61
pubmed: 25568350
Blood. 2012 Nov 29;120(23):4517-20
pubmed: 23043073
Blood. 1977 Sep;50(3):351-66
pubmed: 884315
Thromb Haemost. 2001 Mar;85(3):560
pubmed: 11307831
Blood. 2018 Mar 1;131(9):1022-1031
pubmed: 29246900
Blood. 2012 Nov 29;120(23):4521-3
pubmed: 22919027
Hum Gene Ther. 2011 Nov;22(11):1389-401
pubmed: 21476868
HSS J. 2010 Feb;6(1):37-42
pubmed: 19921342
Br Med J. 1952 Dec 27;2(4799):1378-82
pubmed: 12997790
N Engl J Med. 2011 Dec 22;365(25):2357-65
pubmed: 22149959
JAMA. 2018 Feb 13;319(6):539
pubmed: 29450507
J Hepatol. 2016 Oct;65(4):776-783
pubmed: 27212246
N Engl J Med. 2014 Nov 20;371(21):1994-2004
pubmed: 25409372
Hum Gene Ther. 2015 Feb;26(2):69-81
pubmed: 25419787
N Engl J Med. 2017 Dec 7;377(23):2215-2227
pubmed: 29211678
Mol Ther. 2014 Mar;22(3):567-574
pubmed: 23941813
Blood. 2000 Jun 15;95(12):3678-82
pubmed: 10845896
Thromb Haemost. 2011 Sep;106(3):398-404
pubmed: 21833450
N Engl J Med. 2017 Dec 28;377(26):2519-2530
pubmed: 29224506
Nat Med. 2006 Mar;12(3):342-7
pubmed: 16474400
Proc Natl Acad Sci U S A. 1989 Dec;86(24):10095-9
pubmed: 2481310
Br J Haematol. 2007 Mar;136(6):777-87
pubmed: 17341263