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Titolo:
Gene therapy for cystic fibrosis
Autore:
Boyd, AC;
Indirizzi:
Univ Edinburgh, Western Gen Hosp, Mol Med Ctr, Dept Med Sci,Med Genet Sect, Edinburgh EH4 2XU, Midlothian, Scotland Univ Edinburgh Edinburgh Midlothian Scotland EH4 2XU Midlothian, Scotland
Titolo Testata:
EXPERT OPINION ON THERAPEUTIC PATENTS
fascicolo: 1, volume: 11, anno: 2001,
pagine: 1 - 15
SICI:
1354-3776(200101)11:1<1:GTFCF>2.0.ZU;2-C
Fonte:
ISI
Lingua:
ENG
Soggetto:
LIPID-PDNA COMPLEXES; EPITHELIA IN-VITRO; AIRWAY EPITHELIA; MAMMALIAN-CELLS; PLASMID DNA; RECOMBINANT ADENOVIRUS; TRANSGENE EXPRESSION; BIOLOGICAL EFFICACY; RETROVIRAL VECTORS; VIRUS VECTORS;
Keywords:
cystic fibrosis (CF); cystic fibrosis transmembrane conductance; regulator (CFTR); expression vectors; gene therapy; lipofection; lung epithelium; viral vehicles;
Tipo documento:
Review
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
141
Recensione:
Indirizzi per estratti:
Indirizzo: Boyd, AC Univ Edinburgh, Western Gen Hosp, Mol Med Ctr, Dept Med Sci,Med Genet Sect, Crewe Rd, Edinburgh EH4 2XU, Midlothian, Scotland Univ EdinburghCrewe Rd Edinburgh Midlothian Scotland EH4 2XU and
Citazione:
A.C. Boyd, "Gene therapy for cystic fibrosis", EXPERT OP T, 11(1), 2001, pp. 1-15

Abstract

After a decade of intensive research, effective gene therapy for cystic fibrosis (CF) remains an elusive goal. There have, however, been tremendous advances in our understanding of the basic processes of CF lung disease and in the development of gene therapy protocols, as reflected in the number and diversity of relevant patents issued. A selection of pertinent patents (with a bins towards those granted by the WIPO) is reviewed. Solutions to thekey challenge of efficiently and safely delivering therapeutic genes to the respiratory epithelium are actively being sought. A new generation of vehicles that more effectively target cells while minimising immune responses is emerging. Vehicles and vectors that promote extended transgene persistence and expression are being developed to outcome the problems of short-termexpression that characterised early clinical trials. There is ample cause for continued optimism that CF gene therapy will become a clinical reality through incremental improvements in all stages of the therapeutic process from Formulation to expression.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 07/07/20 alle ore 12:28:02