Detalhe da pesquisa
1.
AAV mediated repression of Neat1 lncRNA combined with F8 gene augmentation mitigates pathological mediators of joint disease in haemophilia.
J Cell Mol Med
; 28(11): e18460, 2024 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-38864710
2.
Evaluation of the Anticancer Activity of pH-Sensitive Polyketal Nanoparticles for Acute Myeloid Leukemia.
Mol Pharm
; 18(5): 2015-2031, 2021 05 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33780253
3.
Gene Therapy: Contest between Adeno-Associated Virus and Host Cells and the Impact of UFMylation.
Mol Pharm
; 17(10): 3649-3653, 2020 10 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-32857512
4.
An efficient method to generate xenograft tumor models of acute myeloid leukemia and hepatocellular carcinoma in adult zebrafish.
Blood Cells Mol Dis
; 75: 48-55, 2019 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-30616104
5.
A CD33 Antigen-Targeted AAV6 Vector Expressing an Inducible Caspase-9 Suicide Gene Is Therapeutic in a Xenotransplantation Model of Acute Myeloid Leukemia.
Bioconjug Chem
; 30(9): 2404-2416, 2019 09 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-31436412
6.
Combination Suicide Gene Delivery with an Adeno-Associated Virus Vector Encoding Inducible Caspase-9 and a Chemical Inducer of Dimerization Is Effective in a Xenotransplantation Model of Hepatocellular Carcinoma.
Bioconjug Chem
; 30(6): 1754-1762, 2019 06 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-31181889
7.
Molecular Engineering of Adeno-Associated Virus Capsid Improves Its Therapeutic Gene Transfer in Murine Models of Hemophilia and Retinal Degeneration.
Mol Pharm
; 16(11): 4738-4750, 2019 11 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-31596095
8.
Potential role of long non-coding RNA H19 and Neat1 in haemophilic arthropathy.
J Cell Mol Med
; 27(12): 1745-1749, 2023 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-37183540
9.
Infectivity of adeno-associated virus serotypes in mouse testis.
BMC Biotechnol
; 18(1): 70, 2018 11 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-30384832
10.
Synergistic inhibition of PARP-1 and NF-κB signaling downregulates immune response against recombinant AAV2 vectors during hepatic gene therapy.
Eur J Immunol
; 46(1): 154-66, 2016 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-26443873
11.
Enhanced transgene expression from recombinant single-stranded D-sequence-substituted adeno-associated virus vectors in human cell lines in vitro and in murine hepatocytes in vivo.
J Virol
; 89(2): 952-61, 2015 Jan 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-25355884
12.
MicroRNA-15b Modulates Molecular Mediators of Blood Induced Arthropathy in Hemophilia Mice.
Int J Mol Sci
; 17(4): 492, 2016 Apr 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-27070581
13.
Adeno-associated virus (AAV) vectors in gene therapy: immune challenges and strategies to circumvent them.
Rev Med Virol
; 23(6): 399-413, 2013 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-24023004
14.
Activation of the NF-kappaB pathway by adeno-associated virus (AAV) vectors and its implications in immune response and gene therapy.
Proc Natl Acad Sci U S A
; 108(9): 3743-8, 2011 Mar 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-21321191
15.
AAV mediated genome engineering with a bypass coagulation factor alleviates the bleeding phenotype in a murine model of hemophilia B.
Thromb Res
; 238: 151-160, 2024 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-38718473
16.
Generation of two induced pluripotent stem cell lines (LVPEIi004-A and LVPEIi005-A) from probands with Leber Congenital Amaurosis 2 (LCA2) and harboring mutations in RPE65.
Stem Cell Res
; 77: 103413, 2024 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-38631180
17.
Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo.
Cytotherapy
; 15(8): 986-98, 2013 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-23830234
18.
Inducible caspase 9-mediated suicide gene therapy using AAV6 vectors in a murine model of breast cancer.
Mol Ther Methods Clin Dev
; 31: 101166, 2023 Dec 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38149057
19.
Targeted delivery of miR125a-5p and human Factor VIII attenuates molecular mediators of hemophilic arthropathy.
Thromb Res
; 231: 8-16, 2023 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-37741049
20.
Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina.
Mol Ther
; 19(2): 293-301, 2011 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-21045809