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.
Enhanced HbF reactivation by multiplex mutagenesis of thalassemic CD34+ cells in vitro and in vivo.
Blood;
138(17): 1540-1553, 2021 10 28.
Artigo
em Inglês
| MEDLINE
| ID: mdl-34086867
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.
Vaccinated and Convalescent Donor-Derived Severe Acute Respiratory Syndrome Coronavirus 2-Specific T Cells as Adoptive Immunotherapy for High-Risk Coronavirus Disease 2019 Patients.
Clin Infect Dis;
73(11): 2073-2082, 2021 12 06.
Artigo
em Inglês
| MEDLINE
| ID: mdl-33905481
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.
The establishment of mitotic errors-driven senescence depends on autophagy.
Redox Biol;
62: 102701, 2023 06.
Artigo
em Inglês
| MEDLINE
| ID: mdl-37094517
7.
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
8.
SARS-CoV-2-specific T cell therapy for severe COVID-19: a randomized phase 1/2 trial.
Nat Med;
29(8): 2019-2029, 2023 08.
Artigo
em Inglês
| MEDLINE
| ID: mdl-37460756
9.
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
10.
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
11.
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
12.
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
13.
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
14.
"Cerberus" T Cells: A Glucocorticoid-Resistant, Multi-Pathogen Specific T Cell Product to Fight Infections in Severely Immunocompromised Patients.
Front Immunol;
11: 608701, 2020.
Artigo
em Inglês
| MEDLINE
| ID: mdl-33537032
15.
In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia.
J Clin Invest;
129(2): 598-615, 2019 02 01.
Artigo
em Inglês
| MEDLINE
| ID: mdl-30422819