Detalhe da pesquisa
1.
In vivo HSC prime editing rescues sickle cell disease in a mouse model.
Blood
; 141(17): 2085-2099, 2023 04 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-36800642
2.
Stable HIV decoy receptor expression after in vivo HSC transduction in mice and NHPs: Safety and efficacy in protection from SHIV.
Mol Ther
; 31(4): 1059-1073, 2023 04 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-36760126
3.
In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model.
Mol Ther
; 29(2): 822-837, 2021 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-32949495
4.
Reactivation of γ-globin in adult ß-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing.
Blood
; 131(26): 2915-2928, 2018 06 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-29789357
5.
Targeted Integration and High-Level Transgene Expression in AAVS1 Transgenic Mice after In Vivo HSC Transduction with HDAd5/35++ Vectors.
Mol Ther
; 27(12): 2195-2212, 2019 12 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-31494053
6.
Stable HIV decoy receptor expression after in vivo HSC transduction in mice and NHPs: Safety and efficacy in protection from SHIV.
Mol Ther
; 31(4): 1188, 2023 Apr 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-36882062
7.
Studies on the Interaction of Tumor-Derived HD5 Alpha Defensins with Adenoviruses and Implications for Oncolytic Adenovirus Therapy.
J Virol
; 91(6)2017 03 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-28077642
8.
CYR61 (CCN1) overexpression induces lung injury in mice.
Am J Physiol Lung Cell Mol Physiol
; 308(8): L759-65, 2015 Apr 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-25713320
9.
HDAd6/35++ - A new helper-dependent adenovirus vector platform for in vivo transduction of hematopoietic stem cells.
Mol Ther Methods Clin Dev
; 29: 213-226, 2023 Jun 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-37081854
10.
Fas-deficient mice have impaired alveolar neutrophil recruitment and decreased expression of anti-KC autoantibody:KC complexes in a model of acute lung injury.
Respir Res
; 13: 91, 2012 Oct 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-23043753
11.
In vivo HSC transduction in rhesus macaques with an HDAd5/3+ vector targeting desmoglein 2 and transiently overexpressing cxcr4.
Blood Adv
; 6(15): 4360-4372, 2022 08 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-35679480
12.
In Vivo Hematopoietic Stem Cell Gene Therapy for SARS-CoV2 Infection Using a Decoy Receptor.
Hum Gene Ther
; 33(7-8): 389-403, 2022 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-35057635
13.
Translational development of a tumor junction opening technology.
Sci Rep
; 12(1): 7753, 2022 05 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-35562182
14.
In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies.
JCI Insight
; 7(19)2022 10 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-36006707
15.
Erratum: Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors.
Mol Ther Methods Clin Dev
; 25: 533, 2022 Jun 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-35662812
16.
Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors.
Mol Ther Methods Clin Dev
; 24: 127-141, 2022 Mar 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-35036470
17.
In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in ß-YAC mice.
Blood Adv
; 5(4): 1122-1135, 2021 02 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-33620431
18.
Single-dose MGTA-145/plerixafor leads to efficient mobilization and in vivo transduction of HSCs with thalassemia correction in mice.
Blood Adv
; 5(5): 1239-1249, 2021 03 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-33646305
19.
Curative in vivo hematopoietic stem cell gene therapy of murine thalassemia using large regulatory elements.
JCI Insight
; 5(16)2020 08 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-32814708
20.
High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells.
Blood Adv
; 3(19): 2883-2894, 2019 10 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-31585952