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.
Computer-aided design, synthesis and validation of 2-phenylquinazolinone fragments as CDK9 inhibitors with anti-HIV-1 Tat-mediated transcription activity.
ChemMedChem
; 8(12): 1941-53, 2013 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-24150998
3.
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