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Titolo:
Perspectives on gene therapy for cystic fibrosis airway disease
Autore:
Bigger, B; Coutelle, C;
Indirizzi:
Univ London Imperial Coll Sci Technol & Med, Div Cell & Mol Genet, Cyst Fibrosis Gene Therapy Res Grp, London SW7 2AZ, England Univ London Imperial Coll Sci Technol & Med London England SW7 2AZ gland
Titolo Testata:
BIODRUGS
fascicolo: 9, volume: 15, anno: 2001,
pagine: 615 - 634
SICI:
1173-8804(2001)15:9<615:POGTFC>2.0.ZU;2-I
Fonte:
ISI
Lingua:
ENG
Soggetto:
TRANSMEMBRANE CONDUCTANCE REGULATOR; ADENOVIRUS-MEDIATED TRANSFER; NASAL POTENTIAL DIFFERENCE; ADENOASSOCIATED VIRUS VECTOR; HUMAN CFTR CDNA; IN-VIVO; NONHUMAN-PRIMATES; ION-TRANSPORT; RECOMBINANT ADENOVIRUS; INFLAMMATORY RESPONSE;
Tipo documento:
Review
Natura:
Periodico
Settore Disciplinare:
Clinical Medicine
Citazioni:
156
Recensione:
Indirizzi per estratti:
Indirizzo: Coutelle, C Univ London Imperial Coll Sci Technol & Med, Div Cell & Mol Genet, Cyst Fibrosis Gene Therapy Res Grp, Sir Alexander Fleming Bldg, LondonSW7 2AZ, England Univ London Imperial Coll Sci Technol & Med Sir AlexanderFleming Bldg London England SW7 2AZ
Citazione:
B. Bigger e C. Coutelle, "Perspectives on gene therapy for cystic fibrosis airway disease", BIODRUGS, 15(9), 2001, pp. 615-634

Abstract

Since the discovery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene nearly 12 years ago, cystic fibrosis (CF) has become one of the most intensively investigated monogenetic disorders considered approachable by gene therapy. This has resulted in over 20 clinical trials currently under way. concluded or awaiting approval. Despite the initial promise of gene therapy for CF, and the demonstration of successful gene transfer to the nose and airways of individuals, it has not so far been as effective as initially projected. Here we discuss the rationale behind CF gene therapy and dissect the vast array of literature representing the work that ultimately brought about the current phase I/II clinical trials. In the context of human trials, we review the limitations of current vector systems for CFgene therapy. We come to the conclusion that at present none of the application methods and vector systems are able to achieve the level and persistence of CFTR gene expression in the affected epithelia of CF patients that is required for therapeutic success. We also outline the challenges that must be overcome and describe some of the novel approaches to be taken in order to attain the curative therapy that was originally envisaged for this disease.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 19/01/20 alle ore 20:29:00