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Titolo:
Development of novel formulations that enhance adenoviral-mediated gene expression in the lung in vitro and in vivo
Autore:
Croyle, MA; Cheng, X; Sandhu, A; Wilson, JM;
Indirizzi:
Univ Texas, Coll Pharm, Div Pharmaceut, Austin, TX 78712 USA Univ Texas Austin TX USA 78712 harm, Div Pharmaceut, Austin, TX 78712 USA Univ Penn, Inst Human Gene Therapy, Philadelphia, PA 19104 USA Univ Penn Philadelphia PA USA 19104 e Therapy, Philadelphia, PA 19104 USA Univ Penn, Dept Mol & Cellular Engn, Philadelphia, PA 19104 USA Univ PennPhiladelphia PA USA 19104 ular Engn, Philadelphia, PA 19104 USA
Titolo Testata:
MOLECULAR THERAPY
fascicolo: 1, volume: 4, anno: 2001,
pagine: 22 - 28
SICI:
1525-0016(200107)4:1<22:DONFTE>2.0.ZU;2-5
Fonte:
ISI
Lingua:
ENG
Soggetto:
CONDUCTANCE REGULATOR GENE; NASAL DELIVERY SYSTEM; CYSTIC-FIBROSIS; IN-VIVO; AIRWAY EPITHELIA; NONHUMAN-PRIMATES; MOUSE LUNG; CFTR CDNA; COMPLEXES; CHITOSAN;
Keywords:
adenovirus; formulation; lung; gene therapy; stability;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
43
Recensione:
Indirizzi per estratti:
Indirizzo: Croyle, MA Univ Texas, Coll Pharm, Div Pharmaceut, Austin, TX 78712 USA Univ Texas Austin TX USA 78712 harmaceut, Austin, TX 78712 USA
Citazione:
M.A. Croyle et al., "Development of novel formulations that enhance adenoviral-mediated gene expression in the lung in vitro and in vivo", MOL THER, 4(1), 2001, pp. 22-28

Abstract

Despite remarkable progress in the development of both viral and non-viralgene delivery vectors for cystic fibrosis therapy, low efficiency of gene transfer to the airway epithelium is a major obstacle to clinical application. Here we develop formulations that enhance cellular absorption of adenoviral vectors. We selected excipients from a panel of pharmaceutically acceptable compounds known to enhance drug absorption. Transduction efficiency of the virus in the presence of each ingredient was assessed in vitro and invivo. Mannitol and chitosan substantially enhanced transduction efficiencyin vitro and augmented expression in vivo by 4 and 8 log units, respectively. The most successful formulation (a blend of sucrose, mannitol, and Pluronic F68) transduced 100% of an A549 cell population in vitro and produced areas of intense gene expression in both large and small airways in vivo with minimal toxicity. Dose response studies also indicate that when placed in this formulation, the viral dose can be lowered by 1/2 log while maintaining superior levels of transgene expression. This formulation also enhancedthe physical stability of the virus. No significant loss in titer was detected from a lyophilized formulation after storage at 25 degreesC for 30 days.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 19/01/20 alle ore 20:43:38