Detalhe da pesquisa
1.
Genomic investigations of unexplained acute hepatitis in children.
Nature
; 617(7961): 564-573, 2023 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-36996872
2.
Hematopoietic stem cell gene editing rescues B-cell development in X-linked agammaglobulinemia.
J Allergy Clin Immunol
; 2024 Mar 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-38479630
3.
Lentiviral gene therapy rescues p47phox chronic granulomatous disease and the ability to fight Salmonella infection in mice.
Gene Ther
; 27(9): 459-469, 2020 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-32533104
4.
Pulmonary metastatic colonisation and granulomas in NOX2-deficient mice.
J Pathol
; 246(3): 300-310, 2018 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-30062795
5.
Myelodysplasia and transgene inactivation in X-CGD-γ retroviral gene therapy: The usual suspects and new players.
Mol Ther
; 31(12): 3367-3368, 2023 Dec 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-37980903
6.
Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions.
J Allergy Clin Immunol
; 138(1): 219-228.e9, 2016 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-26853280
7.
EROS/CYBC1 mutations: Decreased NADPH oxidase function and chronic granulomatous disease.
J Allergy Clin Immunol
; 143(2): 782-785.e1, 2019 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-30312704
8.
Multidimensional Response Surface Methodology for the Development of a Gene Editing Protocol for p67phox-Deficient Chronic Granulomatous Disease.
Hum Gene Ther
; 35(7-8): 298-312, 2024 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-38062734
9.
A New Chapter on Targeted Gene Insertion for X-CGD: Do Not Skip the Intro(n).
Mol Ther
; 25(2): 307-309, 2017 02 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28109956
10.
BCR-ABL suppresses C/EBPalpha expression through inhibitory action of hnRNP E2.
Nat Genet
; 30(1): 48-58, 2002 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-11753385
11.
Gene Editing in Human Haematopoietic Stem Cells for the Treatment of Primary Immunodeficiencies.
Mol Diagn Ther
; 27(1): 15-28, 2023 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36239917
12.
An improved medium formulation for efficient ex vivo gene editing, expansion and engraftment of hematopoietic stem and progenitor cells.
Mol Ther Methods Clin Dev
; 29: 58-69, 2023 Jun 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-36950452
13.
AAV capsid bioengineering in primary human retina models.
Sci Rep
; 13(1): 21946, 2023 12 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-38081924
14.
Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors.
Hum Gene Ther
; 34(7-8): 273-288, 2023 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-36927149
15.
BCR/ABL activates mdm2 mRNA translation via the La antigen.
Cancer Cell
; 3(2): 145-60, 2003 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-12620409
16.
Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells.
Mol Ther
; 19(1): 122-32, 2011 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-20978475
17.
AAV-p40 Bioengineering Platform for Variant Selection Based on Transgene Expression.
Hum Gene Ther
; 33(11-12): 664-682, 2022 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35297686
18.
EROS is a selective chaperone regulating the phagocyte NADPH oxidase and purinergic signalling.
Elife
; 112022 11 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-36421765
19.
Immunodeficiency, autoimmunity, and increased risk of B cell malignancy in humans with TRAF3 mutations.
Sci Immunol
; 7(74): eabn3800, 2022 08 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-35960817
20.
Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease.
Hum Gene Ther
; 32(17-18): 949-958, 2021 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-33740872