Detalhe da pesquisa
1.
Factor IXa variants resistant to plasma inhibitors enhance clot formation in vivo.
Blood;
141(16): 2022-2032, 2023 04 20.
Artigo
em Inglês
| MEDLINE
| ID: mdl-36724452
2.
Immune complications and their management in inherited and acquired bleeding disorders.
Blood;
140(10): 1075-1085, 2022 09 08.
Artigo
em Inglês
| MEDLINE
| ID: mdl-35793465
3.
Gene Therapy for Inherited Bleeding Disorders.
Semin Thromb Hemost;
47(2): 161-173, 2021 Mar.
Artigo
em Inglês
| MEDLINE
| ID: mdl-33636747
4.
Inhibitors-Recent insights.
Haemophilia;
27 Suppl 3: 28-36, 2021 Feb.
Artigo
em Inglês
| MEDLINE
| ID: mdl-32608138
5.
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
N Engl J Med;
377(23): 2215-2227, 2017 12 07.
Artigo
em Inglês
| MEDLINE
| ID: mdl-29211678
6.
Novel approaches to hemophilia therapy: successes and challenges.
Blood;
130(21): 2251-2256, 2017 11 23.
Artigo
em Inglês
| MEDLINE
| ID: mdl-29018078
7.
Gene therapy for hemophilia: Progress to date and challenges moving forward.
Transfus Apher Sci;
58(5): 602-612, 2019 Oct.
Artigo
em Inglês
| MEDLINE
| ID: mdl-31543256
8.
A Novel Platform for Immune Tolerance Induction in Hemophilia A Mice.
Mol Ther;
25(8): 1815-1830, 2017 08 02.
Artigo
em Inglês
| MEDLINE
| ID: mdl-28552407
9.
AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice.
Blood;
125(10): 1553-61, 2015 Mar 05.
Artigo
em Inglês
| MEDLINE
| ID: mdl-25568350
10.
Gene therapy matures to medicines.
Hum Mol Genet;
28(R1): R1-R2, 2019 10 01.
Artigo
em Inglês
| MEDLINE
| ID: mdl-31595292
11.
Omental implantation of BOECs in hemophilia dogs results in circulating FVIII antigen and a complex immune response.
Blood;
123(26): 4045-53, 2014 Jun 26.
Artigo
em Inglês
| MEDLINE
| ID: mdl-24829206
12.
Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype.
Blood;
121(21): 4396-403, 2013 May 23.
Artigo
em Inglês
| MEDLINE
| ID: mdl-23372167
13.
Stopping bleeding is not enough to FIX hemarthropathy.
Blood;
129(15): 2048-2049, 2017 04 13.
Artigo
em Inglês
| MEDLINE
| ID: mdl-28408422
14.
The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.
Blood;
120(23): 4521-3, 2012 Nov 29.
Artigo
em Inglês
| MEDLINE
| ID: mdl-22919027
15.
AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life.
Mol Ther Methods Clin Dev;
32(1): 101205, 2024 Mar 14.
Artigo
em Inglês
| MEDLINE
| ID: mdl-38374963
16.
Assessing the potential for AAV vector genotoxicity in a murine model.
Blood;
117(12): 3311-9, 2011 Mar 24.
Artigo
em Inglês
| MEDLINE
| ID: mdl-21106988
17.
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.
Nat Med;
12(3): 342-7, 2006 Mar.
Artigo
em Inglês
| MEDLINE
| ID: mdl-16474400
18.
Characteristics of BAY 2599023 in the Current Treatment Landscape of Hemophilia A Gene Therapy.
Curr Gene Ther;
23(2): 81-95, 2023.
Artigo
em Inglês
| MEDLINE
| ID: mdl-36111754
19.
Analysis of vector genome integrations in multicentric lymphoma after AAV gene therapy in a severe hemophilia A dog.
Mol Ther Methods Clin Dev;
31: 101159, 2023 Dec 14.
Artigo
em Inglês
| MEDLINE
| ID: mdl-38094200
20.
X-linked thrombophilia with a mutant factor IX (factor IX Padua).
N Engl J Med;
361(17): 1671-5, 2009 Oct 22.
Artigo
em Inglês
| MEDLINE
| ID: mdl-19846852