Detalhe da pesquisa
1.
Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis.
Blood
; 143(19): 1937-1952, 2024 May 09.
Artigo
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
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
Inglês
| MEDLINE | ID: mdl-36790505
4.
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
N Engl J Med
; 385(21): 1929-1940, 2021 11 18.
Artigo
Inglês
| MEDLINE | ID: mdl-34788506
5.
AAV integration in human hepatocytes.
Mol Ther
; 29(10): 2898-2909, 2021 10 06.
Artigo
Inglês
| MEDLINE | ID: mdl-34461297
6.
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
Inglês
| MEDLINE | ID: mdl-33010230
7.
γ-TRIS: a graph-algorithm for comprehensive identification of vector genomic insertion sites.
Bioinformatics
; 36(5): 1622-1624, 2020 03 01.
Artigo
Inglês
| MEDLINE | ID: mdl-31589304
8.
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
Inglês
| MEDLINE | ID: mdl-31513879
9.
Targeted genome editing in human repopulating haematopoietic stem cells.
Nature
; 510(7504): 235-240, 2014 Jun 12.
Artigo
Inglês
| MEDLINE | ID: mdl-24870228
10.
VISPA2: a scalable pipeline for high-throughput identification and annotation of vector integration sites.
BMC Bioinformatics
; 18(1): 520, 2017 Nov 25.
Artigo
Inglês
| MEDLINE | ID: mdl-29178837
11.
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
Inglês
| MEDLINE | ID: mdl-27289174
12.
Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency.
Mol Ther
; 24(7): 1187-98, 2016 08.
Artigo
Inglês
| MEDLINE | ID: mdl-27138040
13.
adLIMS: a customized open source software that allows bridging clinical and basic molecular research studies.
BMC Bioinformatics
; 16 Suppl 9: S5, 2015.
Artigo
Inglês
| MEDLINE | ID: mdl-26051409
14.
Lentiviral vector-based insertional mutagenesis identifies genes involved in the resistance to targeted anticancer therapies.
Mol Ther
; 22(12): 2056-2068, 2014 Dec.
Artigo
Inglês
| MEDLINE | ID: mdl-25195596
15.
MYO1E mutations and childhood familial focal segmental glomerulosclerosis.
N Engl J Med
; 365(4): 295-306, 2011 Jul 28.
Artigo
Inglês
| MEDLINE | ID: mdl-21756023
16.
A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID.
Nat Commun
; 15(1): 3662, 2024 Apr 30.
Artigo
Inglês
| MEDLINE | ID: mdl-38688902
17.
SNPranker 2.0: a gene-centric data mining tool for diseases associated SNP prioritization in GWAS.
BMC Bioinformatics
; 14 Suppl 1: S9, 2013.
Artigo
Inglês
| MEDLINE | ID: mdl-23369106
18.
Targeted next-generation sequencing appoints c16orf57 as clericuzio-type poikiloderma with neutropenia gene.
Am J Hum Genet
; 86(1): 72-6, 2010 Jan.
Artigo
Inglês
| MEDLINE | ID: mdl-20004881
19.
InCliniGene enables high-throughput and comprehensive in vivo clonal tracking toward clinical genomics data integration.
Database (Oxford)
; 20232023 11 02.
Artigo
Inglês
| MEDLINE | ID: mdl-37935583
20.
Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy.
Nat Commun
; 14(1): 3068, 2023 05 27.
Artigo
Inglês
| MEDLINE | ID: mdl-37244942