Detalhe da pesquisa
1.
Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis.
Blood
; 143(19): 1937-1952, 2024 May 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-38446574
2.
ISAnalytics enables longitudinal and high-throughput clonal tracking studies in hematopoietic stem cell gene therapy applications.
Brief Bioinform
; 24(1)2023 01 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-36545803
3.
Intrathymic AAV delivery results in therapeutic site-specific integration at TCR loci in mice.
Blood
; 141(19): 2316-2329, 2023 05 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-36790505
4.
Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cells.
Mol Ther
; 32(1): 124-139, 2024 Jan 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-37990494
5.
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
N Engl J Med
; 385(21): 1929-1940, 2021 11 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-34788506
6.
Oncogene-induced maladaptive activation of trained immunity in the pathogenesis and treatment of Erdheim-Chester disease.
Blood
; 138(17): 1554-1569, 2021 10 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-34077954
7.
Evaluating the state of the science for adeno-associated virus integration: An integrated perspective.
Mol Ther
; 30(8): 2646-2663, 2022 08 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-35690906
8.
AAV integration in human hepatocytes.
Mol Ther
; 29(10): 2898-2909, 2021 10 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-34461297
9.
Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy.
Mol Ther
; 29(1): 86-102, 2021 01 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-33010230
10.
γ-TRIS: a graph-algorithm for comprehensive identification of vector genomic insertion sites.
Bioinformatics
; 36(5): 1622-1624, 2020 03 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31589304
11.
Intrathymic adeno-associated virus gene transfer rapidly restores thymic function and long-term persistence of gene-corrected T cells.
J Allergy Clin Immunol
; 145(2): 679-697.e5, 2020 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-31513879
12.
Targeted genome editing in human repopulating haematopoietic stem cells.
Nature
; 510(7504): 235-240, 2014 Jun 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-24870228
13.
VISPA2: a scalable pipeline for high-throughput identification and annotation of vector integration sites.
BMC Bioinformatics
; 18(1): 520, 2017 Nov 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-29178837
14.
Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial.
Lancet
; 388(10043): 476-87, 2016 Jul 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-27289174
15.
Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency.
Mol Ther
; 24(7): 1187-98, 2016 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-27138040
16.
Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy.
Hum Mol Genet
; 23(12): 3250-68, 2014 Jun 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-24463623
17.
Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer.
Nat Methods
; 10(2): 155-61, 2013 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-23314173
18.
Cyclosporin a and rapamycin relieve distinct lentiviral restriction blocks in hematopoietic stem and progenitor cells.
Mol Ther
; 23(2): 352-62, 2015 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-25270076
19.
adLIMS: a customized open source software that allows bridging clinical and basic molecular research studies.
BMC Bioinformatics
; 16 Suppl 9: S5, 2015.
Artigo
em Inglês
| MEDLINE | ID: mdl-26051409
20.
Wiskott-Aldrich syndrome protein deficiency in natural killer and dendritic cells affects antitumor immunity.
Eur J Immunol
; 44(4): 1039-45, 2014 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-24338698