Detalhe da pesquisa
1.
Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua.
Blood
; 137(21): 2902-2906, 2021 05 27.
Artigo
Inglês
| MEDLINE | ID: mdl-33735915
2.
Universal allogeneic CAR T cells engineered with Sleeping Beauty transposons and CRISPR-CAS9 for cancer immunotherapy.
Mol Ther
; 30(10): 3155-3175, 2022 10 05.
Artigo
Inglês
| MEDLINE | ID: mdl-35711141
3.
Comprehensive transcriptome-wide analysis of spliceopathy correction of myotonic dystrophy using CRISPR-Cas9 in iPSCs-derived cardiomyocytes.
Mol Ther
; 30(1): 75-91, 2022 01 05.
Artigo
Inglês
| MEDLINE | ID: mdl-34371182
4.
Distinct transduction of muscle tissue in mice after systemic delivery of AAVpo1 vectors.
Gene Ther
; 27(3-4): 170-179, 2020 04.
Artigo
Inglês
| MEDLINE | ID: mdl-31624368
5.
Towards a global multidisciplinary consensus framework on haemophilia gene therapy: Report of the 2nd World Federation of Haemophilia Gene Therapy Round Table.
Haemophilia
; 26(3): 443-449, 2020 May.
Artigo
Inglês
| MEDLINE | ID: mdl-32202382
6.
Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells.
Nucleic Acids Res
; 46(16): 8275-8298, 2018 09 19.
Artigo
Inglês
| MEDLINE | ID: mdl-29947794
7.
AAV9 delivered bispecific nanobody attenuates amyloid burden in the gelsolin amyloidosis mouse model.
Hum Mol Genet
; 26(7): 1353-1364, 2017 04 01.
Artigo
Inglês
| MEDLINE | ID: mdl-28334940
8.
First hemophilia B gene therapy approved: More than two decades in the making.
Mol Ther
; 31(1): 1-2, 2023 01 04.
Artigo
Inglês
| MEDLINE | ID: mdl-36528029
9.
Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9.
Mol Ther
; 26(5): 1241-1254, 2018 05 02.
Artigo
Inglês
| MEDLINE | ID: mdl-29599079
10.
Hemophilia "A" gene therapy: Lost in translation.
Mol Ther
; 30(12): 3508-3509, 2022 12 07.
Artigo
Inglês
| MEDLINE | ID: mdl-36417911
11.
First conditional marketing authorization approval in the European Union for hemophilia "A" gene therapy.
Mol Ther
; 30(11): 3335-3336, 2022 11 02.
Artigo
Inglês
| MEDLINE | ID: mdl-36261044
12.
A Novel Platform for Immune Tolerance Induction in Hemophilia A Mice.
Mol Ther
; 25(8): 1815-1830, 2017 08 02.
Artigo
Inglês
| MEDLINE | ID: mdl-28552407
13.
piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.
Nucleic Acids Res
; 44(2): 744-60, 2016 Jan 29.
Artigo
Inglês
| MEDLINE | ID: mdl-26682797
14.
Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy.
Blood
; 123(20): 3195-9, 2014 May 15.
Artigo
Inglês
| MEDLINE | ID: mdl-24637359
15.
Immunology of Gene and Cell Therapy.
Mol Ther
; 28(3): 691-692, 2020 03 04.
Artigo
Inglês
| MEDLINE | ID: mdl-32023479
16.
Genome-wide computational analysis reveals cardiomyocyte-specific transcriptional Cis-regulatory motifs that enable efficient cardiac gene therapy.
Mol Ther
; 23(1): 43-52, 2015 Jan.
Artigo
Inglês
| MEDLINE | ID: mdl-25195597
17.
Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver.
Hepatology
; 60(3): 1035-43, 2014 Sep.
Artigo
Inglês
| MEDLINE | ID: mdl-24585515
18.
Targeting endothelial cells by gene therapy.
Blood
; 122(12): 1993-4, 2013 Sep 19.
Artigo
Inglês
| MEDLINE | ID: mdl-24052537
19.
Getting Into the Rhythm With CRISPR.
Circ Res
; 123(8): 928-930, 2018 09 28.
Artigo
Inglês
| MEDLINE | ID: mdl-30355043
20.
Hyperactive piggyBac transposons for sustained and robust liver-targeted gene therapy.
Mol Ther
; 22(9): 1614-24, 2014 Sep.
Artigo
Inglês
| MEDLINE | ID: mdl-25034357