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Titolo:
Functional correction of Fanconi anemia group C hematopoietic cells by theuse of a novel lentiviral vector
Autore:
Yamada, K; Olsen, JC; Patel, M; Rao, KW; Walsh, CE;
Indirizzi:
Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC 27599 USA Univ N Carolina Chapel Hill NC USA 27599 y Ctr, Chapel Hill, NC 27599 USA Univ N Carolina, Cyst Fibrosis Pulm Res & Treatment Ctr, Chapel Hill, NC 27599 USA Univ N Carolina Chapel Hill NC USA 27599 t Ctr, Chapel Hill, NC 27599 USA Univ N Carolina, Dept Pathol, Chapel Hill, NC 27599 USA Univ N Carolina Chapel Hill NC USA 27599 athol, Chapel Hill, NC 27599 USA Univ N Carolina, Dept Med, Chapel Hill, NC 27599 USA Univ N Carolina Chapel Hill NC USA 27599 t Med, Chapel Hill, NC 27599 USA
Titolo Testata:
MOLECULAR THERAPY
fascicolo: 4, volume: 3, anno: 2001,
pagine: 485 - 490
SICI:
1525-0016(200104)3:4<485:FCOFAG>2.0.ZU;2-M
Fonte:
ISI
Lingua:
ENG
Soggetto:
HUMAN-IMMUNODEFICIENCY-VIRUS; EFFICIENCY GENE-TRANSFER; MURINE LEUKEMIA-VIRUS; NONDIVIDING CELLS; CD34(+) CELLS; STABLE TRANSDUCTION; RETROVIRAL VECTORS; VIRAL REPLICATION; STEM-CELLS; HIGH-TITER;
Keywords:
E1AV vector; lentivirus; Fanconi anemia group C (FANCC);
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
40
Recensione:
Indirizzi per estratti:
Indirizzo: Walsh, CE Room 7101,Thurston Bldg,CB 7352, Chapel Hill, NC 27599 USA Room7101,Thurston Bldg,CB 7352 Chapel Hill NC USA 27599 99 USA
Citazione:
K. Yamada et al., "Functional correction of Fanconi anemia group C hematopoietic cells by theuse of a novel lentiviral vector", MOL THER, 3(4), 2001, pp. 485-490

Abstract

Lentiviral vectors transduce nondividing hematopoietic cells more efficiently than other currently available vector systems. Here we report the results of human hematopoietic cell gene transfer using lentiviral vectors basedupon human immunodeficiency virus (HIV-1) and equine infectious anemia virus (EIAV). EIAV is a nonprimate lentivirus and is severely restricted in its host range to horses and closely related equines. We employed the EIAV vector system to test for gene transfer to human Fanconi anemia (FA) hematopoietic cells by comparison with HIV-1- and Moloney murine leukemia virus-based systems. Fanconi anemia is characterized by bone marrow failure secondary to stem cell dysfunction. Fanconi anemia group C EBV-transformed lymphoblasts were transduced with all three viral vectors. Phenotypic correction ofFA cells, as measured by mitomycin C drug resistance, was observed with a similar efficiency in all vector systems. This is the first description of lentiviral correction of FA cells and suggests that lentiviral vectors may be useful for FA gene transfer.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 28/02/20 alle ore 12:44:24