Detalhe da pesquisa
1.
Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease.
Cytotherapy
; 19(9): 1096-1112, 2017 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-28733131
2.
FAAP20: a novel ubiquitin-binding FA nuclear core-complex protein required for functional integrity of the FA-BRCA DNA repair pathway.
Blood
; 119(14): 3285-94, 2012 Apr 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-22343915
3.
Overcoming reprogramming resistance of Fanconi anemia cells.
Blood
; 119(23): 5449-57, 2012 Jun 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-22371882
4.
Correct mRNA processing at a mutant TT splice donor in FANCC ameliorates the clinical phenotype in patients and is enhanced by delivery of suppressor U1 snRNAs.
Am J Hum Genet
; 87(4): 480-93, 2010 Oct 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-20869034
5.
Impaired FANCD2 monoubiquitination and hypersensitivity to camptothecin uniquely characterize Fanconi anemia complementation group M.
Blood
; 114(1): 174-80, 2009 Jul 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-19423727
6.
Interlaboratory assessment of a novel colony-forming unit assay: a multicenter study by the cellular team of Biomedical Excellence for Safer Transfusion (BEST) collaborative.
Transfusion
; 51(9): 2001-5, 2011 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-21569039
7.
Database setup for preclinical studies of gene-modified hematopoiesis.
Methods Mol Biol
; 506: 467-76, 2009.
Artigo
em Inglês
| MEDLINE | ID: mdl-19110645
8.
Copy number determination of genetically-modified hematopoietic stem cells.
Methods Mol Biol
; 506: 281-98, 2009.
Artigo
em Inglês
| MEDLINE | ID: mdl-19110633
9.
Physiological promoters reduce the genotoxic risk of integrating gene vectors.
Mol Ther
; 16(4): 718-25, 2008 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-18334985
10.
Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.
Mol Ther
; 16(3): 590-8, 2008 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-18180772
11.
Insertional mutagenesis by replication-deficient retroviral vectors encoding the large T oncogene.
Ann N Y Acad Sci
; 1106: 95-113, 2007 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-17395733
12.
Molecular, phenotypic, and sample-associated data to describe pluripotent stem cell lines and derivatives.
Sci Data
; 4: 170030, 2017 03 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-28350385
13.
Cell and virus genetics at the roots of gene therapy, retrovirology, and hematopoietic stem cell biology: Wolfram Ostertag (1937-2010).
Hum Gene Ther
; 21(11): 1501-3, 2010 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-21091034