Catalogo Articoli (Spogli Riviste)

OPAC HELP

Titolo:
A promising genetic approach to the treatment of beta-thalassemia
Autore:
May, C; Sadelain, M;
Indirizzi:
Mem Sloan Kettering Canc Ctr, Lab Gene Transfer & Gene Express, New York, NY 10021 USA Mem Sloan Kettering Canc Ctr New York NY USA 10021 New York, NY 10021 USA
Titolo Testata:
TRENDS IN CARDIOVASCULAR MEDICINE
fascicolo: 7, volume: 11, anno: 2001,
pagine: 276 - 280
SICI:
1050-1738(200110)11:7<276:APGATT>2.0.ZU;2-Z
Fonte:
ISI
Lingua:
ENG
Soggetto:
LOCUS-CONTROL REGION; HEMATOPOIETIC STEM-CELLS; MURINE LEUKEMIA-VIRUS; GLOBIN GENE; LENTIVIRAL VECTORS; RETROVIRAL VECTOR; IN-VIVO; LONG; TRANSDUCTION; EXPRESSION;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
29
Recensione:
Indirizzi per estratti:
Indirizzo: May, C Mem Sloan Kettering Canc Ctr, Lab Gene Transfer & Gene Express, Box182, New York, NY 10021 USA Mem Sloan Kettering Canc Ctr Box 182 New York NY USA 10021 0021 USA
Citazione:
C. May e M. Sadelain, "A promising genetic approach to the treatment of beta-thalassemia", TREND CARD, 11(7), 2001, pp. 276-280

Abstract

The stable introduction of a functional gene into autologous stem cells isa potentially powerful approach to treat a number of inherited or acquireddiseases. One challenge facing this approach is to express adequate levelsof the therapeutic transgene in a regulated and sustained fashion, eventually restricting expression to a single lineage developing from the transduced stem cells. Until now, low-level expression, position effects, and transcriptional silencing have hampered the effectiveness of retroviral-mediatedgene transfer. In an effort to overcome these obstacles, we have systematically investigated vectors encoding the human beta -globin gene linked to selected combinations of proximal and distal genetic regulatory elements. Our results demonstrate that with thoughtful vector design one can successfully express long-term, therapeutic levels of virally encoded human beta -globin in the erythroid progeny of hematopoietic stem cells. (Trends Cardiovasc Med 2001;11:276-280). (C) 2001, Elsevier Science Inc.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 06/04/20 alle ore 08:53:32