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Titolo:
Progress in the use of gene transfer methods to treat genetic blood diseases
Autore:
Williams, DA; Smith, FO;
Indirizzi:
Indiana Univ, Sch Med, Howard Hughes Med Inst, Indianapolis, IN 46202 USA Indiana Univ Indianapolis IN USA 46202 d Inst, Indianapolis, IN 46202 USA Indiana Univ, Sch Med, Dept Pediat,Sect Pediat Hematol Oncol, Herman B Wells Ctr Pediat Res, Indianapolis, IN 46202 USA Indiana Univ Indianapolis INUSA 46202 at Res, Indianapolis, IN 46202 USA
Titolo Testata:
HUMAN GENE THERAPY
fascicolo: 15, volume: 11, anno: 2000,
pagine: 2059 - 2066
SICI:
1043-0342(200010)11:15<2059:PITUOG>2.0.ZU;2-5
Fonte:
ISI
Lingua:
ENG
Soggetto:
HEMATOPOIETIC STEM-CELLS; APE LEUKEMIA-VIRUS; MARROW REPOPULATING CELLS; AMPHOTROPIC RETROVIRUS RECEPTOR; CHRONIC GRANULOMATOUS-DISEASE; SITE-SPECIFIC INTEGRATION; AUTOLOGOUS BONE-MARROW; AMINO-ACID TRANSPORTER; EX-VIVO CULTURE; PERIPHERAL-BLOOD;
Tipo documento:
Review
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
75
Recensione:
Indirizzi per estratti:
Indirizzo: Williams, DA 1044 W Walnut,Room 402, Indianapolis, IN 46202 USA 1044 W Walnut,Room 402 Indianapolis IN USA 46202 N 46202 USA
Citazione:
D.A. Williams e F.O. Smith, "Progress in the use of gene transfer methods to treat genetic blood diseases", HUM GENE TH, 11(15), 2000, pp. 2059-2066

Abstract

A report by French physician-scientists suggests a successful application of gene transfer methods in the treatment of two children with severe combined immunodeficiency (SCID) due to defective interleukin 2 receptor common gamma chain. The protocol used in this clinical trial was derived from a number of preclinical and basic studies leading to improved transduction of hematopoietic stem and primitive progenitor cells using retrovirus vectors. These improvements have also been shown to impact transduction of a long-lived progenitor cell in a chemotherapy protocol in cancer patients. The improved results of these human trials come during a period of increased scrutiny and criticism of human gene therapy trials, due, in part, to significanttoxicities in some trials using adenovirus-based vectors. The potential efficacy versus toxicity of phase I trials of human gene therapy is also under question. After many years of research, however, there appears to be realevidence that genetic diseases may be successfully treated by gene transfer techniques. Future clinical studies should be based on continued progressin the understanding of the toxicology of gene delivery systems, vector technology, and target cell manipulation.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 29/11/20 alle ore 00:57:20