Detalhe da pesquisa
1.
Adeno-Associated Virus Gene Therapy for Hemophilia.
Annu Rev Med
; 74: 231-247, 2023 01 27.
Artigo
Inglês
| MEDLINE | ID: mdl-36103998
2.
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.
N Engl J Med
; 385(21): 1961-1973, 2021 11 18.
Artigo
Inglês
| MEDLINE | ID: mdl-34788507
3.
Activated protein C has a regulatory role in factor VIII function.
Blood
; 137(18): 2532-2543, 2021 05 06.
Artigo
Inglês
| MEDLINE | ID: mdl-33512448
4.
Digital haemophilia: Insights into the use of social media for haemophilia care, research and advocacy.
Haemophilia
; 28(2): 247-253, 2022 Mar.
Artigo
Inglês
| MEDLINE | ID: mdl-35167716
5.
Appendix to the Society of Interventional Radiology Consensus Guidelines for the Periprocedural Management of Thrombotic and Bleeding Risk in Patients Undergoing Percutaneous Image-Guided Interventions: Pediatric Considerations.
J Vasc Interv Radiol
; 33(11): 1424-1431, 2022 11.
Artigo
Inglês
| MEDLINE | ID: mdl-35842024
6.
Gene Therapy for Inherited Bleeding Disorders.
Semin Thromb Hemost
; 47(2): 161-173, 2021 Mar.
Artigo
Inglês
| MEDLINE | ID: mdl-33636747
7.
Real-world cost estimates of initiating emicizumab in US patients with haemophilia A.
Haemophilia
; 27(4): 591-598, 2021 Jul.
Artigo
Inglês
| MEDLINE | ID: mdl-34050689
8.
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B.
Mol Ther
; 28(9): 2073-2082, 2020 09 02.
Artigo
Inglês
| MEDLINE | ID: mdl-32559433
9.
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
N Engl J Med
; 377(23): 2215-2227, 2017 12 07.
Artigo
Inglês
| MEDLINE | ID: mdl-29211678
10.
Immune tolerance induction by hepatic gene transfer: First-in-human evidence.
Mol Ther
; 32(4): 863-864, 2024 Apr 03.
Artigo
Inglês
| MEDLINE | ID: mdl-38518767
11.
Novel approaches to hemophilia therapy: successes and challenges.
Blood
; 130(21): 2251-2256, 2017 11 23.
Artigo
Inglês
| MEDLINE | ID: mdl-29018078
12.
MYH9-macrothrombocytopenia caused by a novel variant (E1421K) initially presenting as apparent neonatal alloimmune thrombocytopenia.
Pediatr Blood Cancer
; 65(4)2018 04.
Artigo
Inglês
| MEDLINE | ID: mdl-29286575
13.
Worldwide use of factor IX Padua for hemophilia B gene therapy.
Mol Ther
; 30(7): 2394-2396, 2022 07 06.
Artigo
Inglês
| MEDLINE | ID: mdl-35705095
14.
Andexanet alfa for reversal of rivaroxaban in a child with intracranial hemorrhage.
Pediatr Blood Cancer
; 69(6): e29484, 2022 06.
Artigo
Inglês
| MEDLINE | ID: mdl-34811876
15.
Haemophilia care: the only constant is change.
Br J Haematol
; 194(5): 805-807, 2021 09.
Artigo
Inglês
| MEDLINE | ID: mdl-34340257
16.
Comment on: Bivalirudin during thrombolysis with catheter-directed tPA in a heparin-refractory patient: A case report.
Pediatr Blood Cancer
; 67(12): e28518, 2020 12.
Artigo
Inglês
| MEDLINE | ID: mdl-32618428
17.
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
Inglês
| MEDLINE | ID: mdl-38374963
18.
A review of the rationale for gene therapy for hemophilia A with inhibitors: one-shot tolerance and treatment?
J Thromb Haemost
; 21(11): 3033-3044, 2023 11.
Artigo
Inglês
| MEDLINE | ID: mdl-37225021
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
Inglês
| MEDLINE | ID: mdl-38094200
20.
Immunotherapy targeting B cells and long-lived plasma cells effectively eliminates pre-existing donor-specific allo-antibodies.
Cell Rep Med
; 4(12): 101336, 2023 12 19.
Artigo
Inglês
| MEDLINE | ID: mdl-38118406