Detalhe da pesquisa
1.
The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective.
Blood
; 138(18): 1677-1690, 2021 11 04.
Artigo
Inglês
| MEDLINE | ID: mdl-33895800
2.
Haemophilia, the journey in search of a cure. 1960-2020.
Br J Haematol
; 191(4): 573-578, 2020 11.
Artigo
Inglês
| MEDLINE | ID: mdl-33190257
3.
Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene transfer in early-gestation fetal macaques.
FASEB J
; 33(3): 3954-3967, 2019 03.
Artigo
Inglês
| MEDLINE | ID: mdl-30517034
4.
Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.
Mol Ther
; 26(1): 289-303, 2018 01 03.
Artigo
Inglês
| MEDLINE | ID: mdl-29055620
5.
Recent advances in developing specific therapies for haemophilia.
Br J Haematol
; 181(2): 161-172, 2018 04.
Artigo
Inglês
| MEDLINE | ID: mdl-29359795
6.
Gene Therapy for Hemophilia.
Mol Ther
; 25(5): 1163-1167, 2017 05 03.
Artigo
Inglês
| MEDLINE | ID: mdl-28411016
7.
In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model.
Mol Ther
; 25(8): 1843-1853, 2017 08 02.
Artigo
Inglês
| MEDLINE | ID: mdl-28462816
8.
Polonium-210 poisoning: a first-hand account.
Lancet
; 388(10049): 1075-1080, 2016 Sep 10.
Artigo
Inglês
| MEDLINE | ID: mdl-27461439
9.
Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
N Engl J Med
; 371(21): 1994-2004, 2014 Nov 20.
Artigo
Inglês
| MEDLINE | ID: mdl-25409372
10.
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.
Blood
; 121(17): 3335-44, 2013 Apr 25.
Artigo
Inglês
| MEDLINE | ID: mdl-23426947
11.
Ibrutinib enhances the efficacy of ROR1 bispecific T cell engager mediated cytotoxicity in chronic lymphocytic leukaemia.
Br J Haematol
; 186(2): 380-382, 2019 07.
Artigo
Inglês
| MEDLINE | ID: mdl-30957227
12.
Genetic Targeting of the Albumin Locus to Treat Hemophilia.
N Engl J Med
; 374(13): 1288-90, 2016 Mar 31.
Artigo
Inglês
| MEDLINE | ID: mdl-27028920
13.
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
N Engl J Med
; 365(25): 2357-65, 2011 Dec 22.
Artigo
Inglês
| MEDLINE | ID: mdl-22149959
14.
AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage.
Blood
; 119(4): 957-66, 2012 Jan 26.
Artigo
Inglês
| MEDLINE | ID: mdl-22134170
15.
AAV mediated gene therapy for haemophilia B: From the early attempts to modern trials.
Thromb Res
; 236: 242-249, 2024 Apr.
Artigo
Inglês
| MEDLINE | ID: mdl-38383218
16.
Pre-Existing Immunity to a Nucleic Acid Contaminant-Derived Antigen Mediates Transaminitis and Resultant Diminished Transgene Expression in a Mouse Model of Hepatic Recombinant Adeno-Associated Virus-Mediated Gene Transfer.
Hum Gene Ther
; 2024 Mar 28.
Artigo
Inglês
| MEDLINE | ID: mdl-38420654
17.
Codon optimization of human factor VIII cDNAs leads to high-level expression.
Blood
; 117(3): 798-807, 2011 Jan 20.
Artigo
Inglês
| MEDLINE | ID: mdl-21041718
18.
Differences in wild-type- and R338L-tenase complex formation are at the root of R338L-factor IX assay discrepancies.
Blood Adv
; 7(3): 458-467, 2023 02 14.
Artigo
Inglês
| MEDLINE | ID: mdl-35839077
19.
Transplacental delivery of therapeutic proteins by engineered immunoglobulin G: a step toward perinatal replacement therapy.
J Thromb Haemost
; 21(9): 2405-2417, 2023 09.
Artigo
Inglês
| MEDLINE | ID: mdl-37271431
20.
Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques.
Mol Ther
; 19(11): 1950-60, 2011 Nov.
Artigo
Inglês
| MEDLINE | ID: mdl-21629224