Gene therapy for cystic fibrosis: Which postman, which box?

Thorax

Volumn 53, Issue 3, 1998, Pages 197-199

  Middleton, Peter G ^a   Alton, Eric W F W ^b  

^a WESTMEAD HOSPITAL   (Australia)

^b NATIONAL HEART AND LUNG INSTITUTE   (United Kingdom)

Author keywords

Adenovirus; Cystic fibrosis; Gene therapy; Liposome

Indexed keywords

LIPOSOME; TRANSMEMBRANE CONDUCTANCE REGULATOR; VIRUS VECTOR;

ADENOVIRUS; ARTICLE; CLINICAL TRIAL; CYSTIC FIBROSIS; GENE THERAPY; GENE TRANSFER; HUMAN; INHALATIONAL DRUG ADMINISTRATION; INTRAVENOUS DRUG ADMINISTRATION; PRIORITY JOURNAL;

EID: 0031944810     PISSN: 00406376     EISSN: None     Source Type: Journal    
DOI: 10.1136/thx.53.3.197     Document Type: Article

References (12)

1. Zabner J, Couture LA, Gregory RJ, et al. Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell 1993;75:207-16.
2. Crystal RG, McElvaney NG, Rosenfeld MA, et al. Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nature Genet 1994;8:42-51.
3. Knowles MR, Hohneker KW, Zhou Z, et al. A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis. N Engl J Med 1995;333:823-31.
4. Caplen NJ, Alton EWFW, Middleton PG, et al. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nature Med 1995;1:39-46.
5. Gershon H, Ghirlando R, Guttman SB, et al. Mode of formation and structural features of DNA-cationic liposome complexes used for transfection. Biochemistry 1993; 32:7143-51.
6. Alton EWFW, Middleton PG, Caplen NJ, et al. Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice. Nature Genet 1993;5:135-42.
7. Middleton PG, Caplen NJ, Gao X, et al. Nasal application of the cationic liposome DC-Chol:DOPE does not alter ion transport, lung function or bacterial growth. Ear Respir J 1994;7:442-5.
8. Yang Y, Nunes FA, Berencsi K, et al. Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis. Nature Genet 1994;7:362-9.
9. Middleton PG, Geddes DM, Alton EWFW. Protocols for in vivo measurement of the ion transport defects in cystic fibrosis nasal epithelium. Eur Respir J 1994;7:2050-6.
10. Ferkol T, Perales JC, Eckman E, et al. Gene transfer into the airway epithelium of animals by targeting the polymeric immunoglobulin receptor. J Clin Invest 1995;95:493-502.
11. Johnson LG, Olsen JC, Sarkadi B, et al. Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nature Genet 1992;2:21-5.
12. Dorin JR, Farley R, Webb S, et al. A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction. Gene Therapy 1996;3:797-801.