Detalhe da pesquisa
1.
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.
Lancet
; 399(10322): 372-383, 2022 01 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-35065785
2.
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
3.
Epigenetic regulation of survivin by Bmi1 is cell type specific during corticogenesis and in gliomas.
Stem Cells
; 31(1): 190-202, 2013 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-23132836
4.
Conditional activation of Bmi1 expression regulates self-renewal, apoptosis, and differentiation of neural stem/progenitor cells in vitro and in vivo.
Stem Cells
; 29(4): 700-12, 2011 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-21305672
5.
Toward Reference Intervals of ARSA Activity in the Cerebrospinal Fluid: Implication for the Clinical Practice of Metachromatic Leukodystrophy.
J Appl Lab Med
; 6(2): 354-366, 2021 03 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32910180
6.
Retrieval of vector integration sites from cell-free DNA.
Nat Med
; 27(8): 1458-1470, 2021 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-34140705
7.
Correction to: c-MYC overexpression induces choroid plexus papillomas through a T-cell mediated inflammatory mechanism.
Acta Neuropathol Commun
; 7(1): 179, 2019 Nov 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-31727166
8.
c-MYC overexpression induces choroid plexus papillomas through a T-cell mediated inflammatory mechanism.
Acta Neuropathol Commun
; 7(1): 95, 2019 05 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-31142360
9.
Critical issues for the proper diagnosis of Metachromatic Leukodystrophy.
Gene
; 537(2): 348-51, 2014 Mar 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-24334127
10.
Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy.
Bone Marrow Transplant
; 53(7): 913-917, 2018 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-29379168