Catalogo Articoli (Spogli Riviste)

OPAC HELP

Titolo:
Haemophilias: advances towards genetic engineering replacement therapy
Autore:
Emilien, G; Maloteaux, JM; Penasse, C; Goodeve, A; Casimir, C;
Indirizzi:
Catholic Univ Louvain, Clin Univ St Luc, Pharmacol Lab, B-3000 Louvain, Belgium Catholic Univ Louvain Louvain Belgium B-3000 ab, B-3000 Louvain, Belgium Estab Francais Sang, Versailles, France Estab Francais Sang Versailles France Francais Sang, Versailles, France Royal Hallamshire Hosp, Div Med & Mol Genet, Sheffield S10 2JF, S Yorkshire, England Royal Hallamshire Hosp Sheffield S Yorkshire England S10 2JF ire, England Imperial Coll Sch Med, Dept Haematol, London, England Imperial Coll Sch Med London England ed, Dept Haematol, London, England
Titolo Testata:
CLINICAL AND LABORATORY HAEMATOLOGY
fascicolo: 6, volume: 22, anno: 2000,
pagine: 313 - 323
SICI:
0141-9854(200012)22:6<313:HATGER>2.0.ZU;2-9
Fonte:
ISI
Lingua:
ENG
Soggetto:
HUMAN FACTOR-VIII; HUMAN FACTOR-IX; CLOTTING FACTOR-VIII; ADENOVIRUS-MEDIATED EXPRESSION; ADENOASSOCIATED VIRAL VECTORS; MODIFIED SKIN FIBROBLASTS; RECOMBINANT FACTOR-VIII; COAGULATION-FACTOR-IX; LONG-TERM EXPRESSION; SEVERE HEMOPHILIA-A;
Keywords:
haemophilia; coagulation; factor VIII; factor IX; thrombin; fibrinogen; gene therapy; viral vector;
Tipo documento:
Review
Natura:
Periodico
Settore Disciplinare:
Clinical Medicine
Citazioni:
80
Recensione:
Indirizzi per estratti:
Indirizzo: Emilien, G 127 Rue Henri Prou, F-78340 Les Clayes Sous Bois, France 127 Rue Henri Prou Les Clayes Sous Bois France F-78340 France
Citazione:
G. Emilien et al., "Haemophilias: advances towards genetic engineering replacement therapy", CLIN LAB H, 22(6), 2000, pp. 313-323

Abstract

Both haemophilia A and B are X-linked recessive disorders and therefore occur almost exclusively in males, The genes for both factors VIII and IX have been mapped to the distal end of the long arm of the X chromosome, bands Xq28 and Xq27.1, respectively. The Factor VIII gene comprises 186kb DNA with 9 kb of exon of DNA which encodes an mRNA of nearly 9 kb, The Factor IX gene is 34 kb in length and the essential genetic information is present in eight exons which encode 1.6kb mRNA. in gene therapy, genetic modification of the target cells can be either ex: vivo or in vivo. The advantage of theex vivo approach is that the genetic modification is strictly limited to the isolated cells, In the in vivo approach, the integrity of the target tissue is maintained but the major challenge is to deliver the gene to the target tissue. The use of improved retroviral and adenovirus-based vectors forgene therapy has produced clinically relevant levels of human factor VIII in mice and haemophilic dogs. If further improvements can increase the persistence of expression and decrease the immunological responses, phase I clinical trials in patients can be considered.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 05/12/20 alle ore 01:56:34