Detalhe da pesquisa
1.
Adeno-Associated Virus Gene Therapy for Hemophilia.
Annu Rev Med
; 74: 231-247, 2023 01 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-36103998
2.
Factor VIII trafficking to CD4+ T cells shapes its immunogenicity and requires several types of antigen-presenting cells.
Blood
; 142(3): 290-305, 2023 07 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-37192286
3.
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.
N Engl J Med
; 385(21): 1961-1973, 2021 11 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-34788507
4.
Activated protein C has a regulatory role in factor VIII function.
Blood
; 137(18): 2532-2543, 2021 05 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-33512448
5.
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mol Ther
; 29(2): 464-488, 2021 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33309881
6.
Hemophilia A Gene Therapy - Some Answers, More Questions.
N Engl J Med
; 388(8): 761-763, 2023 02 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-36812440
7.
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
em Inglês
| MEDLINE | ID: mdl-32559433
8.
Total Knee Arthroplasty after Gene Therapy for Hemophilia B.
N Engl J Med
; 387(17): 1622-1624, 2022 10 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-36306204
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
em Inglês
| MEDLINE | ID: mdl-29211678
10.
The national blueprint for future factor VIII inhibitor clinical trials: NHLBI State of the Science (SOS) Workshop on factor VIII inhibitors.
Haemophilia
; 25(4): 581-589, 2019 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-31329364
11.
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
Lancet
; 390(10097): 849-860, 2017 Aug 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-28712537
12.
No CpGs for AAVs?
Blood
; 137(6): 721-723, 2021 02 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-33570612
13.
Haemophilia care: the only constant is change.
Br J Haematol
; 194(5): 805-807, 2021 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-34340257
14.
Maternal nutrient restriction in the ewe from early to midgestation programs reduced steroidogenic enzyme expression and tended to reduce progesterone content of corpora lutea, as well as circulating progesterone in nonpregnant aged female offspring.
Reprod Biol Endocrinol
; 11: 34, 2013 May 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-23656912
15.
Coagulation factor VIII regulates von Willebrand factor homeostasis invivo.
J Thromb Haemost
; 21(12): 3477-3489, 2023 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-37726033
16.
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: facilitating research through infrastructure, workforce, resources and funding.
Expert Rev Hematol
; 16(sup1): 107-127, 2023 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-36920855
17.
Early maternal undernutrition programs increased feed intake, altered glucose metabolism and insulin secretion, and liver function in aged female offspring.
Am J Physiol Regul Integr Comp Physiol
; 302(7): R795-804, 2012 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-22277936
18.
Hemophilia gene therapy: ushering in a new treatment paradigm?
Hematology Am Soc Hematol Educ Program
; 2021(1): 226-233, 2021 12 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-34889378
19.
Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX-Padua.
J Thromb Haemost
; 19(5): 1212-1218, 2021 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-33636038
20.
Different levels of overnutrition and weight gain during pregnancy have differential effects on fetal growth and organ development.
Reprod Biol Endocrinol
; 8: 75, 2010 Jun 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-20576133