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Titolo:
Gene therapy: a 2001 perspective
Autore:
High, KA;
Indirizzi:
Childrens Hosp Philadelphia, Dept Paediat, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia Philadelphia PA USA 19104 lphia, PA 19104 USA
Titolo Testata:
HAEMOPHILIA
, volume: 7, anno: 2001, supplemento:, 1
pagine: 23 - 27
SICI:
1351-8216(200101)7:<23:GTA2P>2.0.ZU;2-6
Fonte:
ISI
Lingua:
ENG
Soggetto:
COAGULATION-FACTOR-IX; DEFICIENT MOUSE MODEL; CANINE HEMOPHILIA-B; FACTOR-VIII; EXPRESSION; VECTOR; VIRUS; MICE; INJECTION; ANTIBODY;
Keywords:
haemophilia A; haemophilia B; gene therapy; retrovirus; AAV; plasmid;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Clinical Medicine
Citazioni:
25
Recensione:
Indirizzi per estratti:
Indirizzo: High, KA Childrens Hosp Philadelphia, Dept Paediat, 3516 Civ Ctr Blvd,310 Abramson Res Ctr, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia 3516 Civ Ctr Blvd,310 Abramson Res Ctr Philadelphia PA USA 19104
Citazione:
K.A. High, "Gene therapy: a 2001 perspective", HAEMOPHILIA, 7, 2001, pp. 23-27

Abstract

In the past year, three clinical trials of gene therapy for haemophilia have been initiated. Years of preclinical studies have culminated in translation of research findings into the clinical arena. It is too early to predict which, if any, of these strategies will show efficacy. This paper will review basic aspects of gene therapy for haemophilia and will briefly outlinecurrent clinical trials. The three clinical trials all share a dose escalation design. The ongoing trial for haemophilia B involves the intramuscularadministration of an adenoassociated virus (AAV) vector expressing human factor IX. In preclinical studies, this strategy has produced therapeutic levels of circulating factor IX in haemophilic mice and dogs.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 02/07/20 alle ore 19:21:52