Utility of the nasal model in gene transfer studies in cystic fibrosis

Rhinology

Volumn 35, Issue 4, 1997, Pages 149-153

  Graham, Scott M ^a,b   Launspach, Janice L ^a  

^a UNIVERSITY OF IOWA   (United States)

^b UNIVERSITY OF IOWA HOSPITALS AND CLINICS   (United States)

Author keywords

Cystic fibrosis; Gene transfer

Indexed keywords

CLINICAL TRIAL; CLONING VECTOR; CYSTIC FIBROSIS; GENE TARGETING; GENE THERAPY; GENE TRANSFER; HUMAN; NONHUMAN; NOSE MUCOSA; REVIEW; TRACHEOBRONCHIAL TREE; TREATMENT OUTCOME;

ADENOVIRIDAE; ADMINISTRATION, INTRANASAL; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS;

EID: 0031415905     PISSN: 03000729     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (22)

1. Blau HM, Springer ML, (1995) Molecular medicine, gene therapy. A novel form of drug delivery. N Engl J Med 1204-1207.
2. Caplan NJ, Alton EWFW, Middleton PG, Dorin JR, Stevenson BJ, Gao X, Durham SR, Jeffery PK, Hodson ME, Coutelle C, Huang L, Porteous DJ, Williamson R, Geddes DM (1995) Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nature Med 1: 39-46.
3. Crystal RG, McElvaney NG, Rosenfeld MA, Chu CS, Mastrangeli A, Hay JG, Brody SL, Jaffe HA, Elissa NT, Danel C (1994) Administration of an adenovirus containing the human CFTR-cDNA to the respiratory tract of individuals with cystic fibrosis. Nature Genetics 8: 42-51.
4. Fitzsimmons SC (1993) The changing epidemiology of cystic fibrosis. J Pediatrics 122: 1-9.
5. Grubb BR, Pickles RJ, Ye H, Yankaskas JR, Vick RN, Engelhardt JF, Wilson JM, Johnson LG, Boucher RC (1994) Inefficient gene transfer by adenovirus vector to cystic fibrosis airway epithelia of mice and humans. Nature 371: 802 806.
6. Hay JG, McElvaney NG, Herena J, Crystal RG (1995) Modification of nasal epithelia potential differences of individuals with cystic fibrosis consequent to local administration of a normal CFTR-cDNA adenovirus gene transfer vector. Human Gene Therapy 6: 1487-1496.
7. Ingels KJ, Nijziel MR, Graamans K, Huizing EH (1994) Influence of cocaine and lidocaine on human nasal cilia. Beat frequency and harmony in vitro. Arch Otolaryngol Head Neck Surg 120: 197-201.
8. Knowles M, Gatzy J, Boucher R (1981) Increased Bioelectric potential difference across respiratory epithelia in cystic fibrosis. N Engl J Med 305: 1489-95.
9. Knowles MR, Hohneker KW, Zhou Z, Olsen JC, Noah TL, Hu PC, Leigh MW, Engelhardt JR, Edwards LJ, Jones KR, Grossman M, Wilson JM, Johnson LG, Boucher RC (1995) A controlled study of adenoviral vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis. N Engl J Med 333: 823-31.
10. Melon J (1968) Contribution a l'étude de l'activité secrétoire de la mugueuse nasalc. Acta Otorhinolaryngol Belg 22: 5216.
11. Quinton PM (1990). Cystic fibrosis, a disease in electrolyte transport. FASEB J 4: 2709-2717.
12. Rich DP, Anderson MP, Gregory RJ, Cheng SH, Paul S, Jefferson DM, McCann JD, Klinger KW, Smith AE, Welsh MJ (1990) Expression of cystic fibrosis transmembrane conductance regulator corrects defective chloride channel regulation in cystic fibrosis airway epithelial cells. Nature 347: 358-363.
13. Rommens JM, Ianuzzi MC, Kerem B, Drumm ML, Melner G, Dean M, Rozmahel R, Cole JL, Kennedy DW, Hidaka N, Zsiga M, Buchwald M, Riordan JR, Tsui LC, Collins FS (1989) Identification of the cystic fibrosis gene: Chromosome walking and jumping. Science 245: 1059-1065.
14. Smith JJ, Travis SM, Greenberg EP, Welsh MJ (1996) Cystic fibrosis airway epithelia fail to kill bacteria because of abnormal airway surface fluid. Cell 85: 229-236.
15. Welsh MJ, Anderson MP, Rich DP, Berger HA, Denning GM, Ostedgaard LS, Sheppard DN, Cheng SH, Gregory RJ, Smith AE (1992) Cystic fibrosis transmembrane conductance regulator: A chloride channel with novel regulation. Neuron 8: 821-829.
16. Welsh MJ, Smith AE, Zabner J, Rich DP, Graham SM, Gregory RJ, Pratt BM, Moscicki RA (1994) Cystic fibrosis gene therapy using an adenovirus vector: In vivo safety and efficacy in nasal epithelium. Human Gene Therapy 5: 209-219.
17. Welsh MJ, Tsui LC, Boat TF, Beaudet AL (1995a) Cystic fibrosis. In: CR Scriver, AL Beaudet, WS Sly and D Valle (Eds.) The Metabolic and Molecular Bases of Inherited Disease, 7th Edition. McGraw-Hill, New York, pp. 3799-3876.
18. Welsh MJ, Zabner J, Graham SM, Smith AE, Moscicki R, Wadsworth S (1995b) Adenovirus-mediated gene transfer for cystic fibrosis: Part A. Safety of dose and repeat administration in the nasal epithelium. Part B. Clinical efficacy in the maxillary sinus. Human Gene Therapy 6: 205-218.
19. Zabner J, Couture LA, Gregory RJ, Graham SM, Smith AE, Welsh MJ (1993) Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell 75: 207-216.
20. Zabner J, Peterson DM, Puga AP, Graham SM, Couture LA, Keyes LD, Lukason MJ, St. George GA, Gregory RJ, Smith AE, Welsh MJ (1994) Safety and efficacy of repetitive adenovirus-mediated transfer of CFTR-cDNA to airway epithelia of primates and cotton rats. Nature Genetics 6: 75-83.
21. Zabner J, Zeiher BG, Friedman E, Welsh MJ (1996a) Adenovirus-mediated gene transfer to ciliated airway epithelia requires prolonged incubation time. J Virol 70: 6994-7003.
22. Zabner J, Ramsey BW, Meeker DP, Aitken ML, Balfour RP, Gibson RL, Launspach J, Moscicki RA, Richards SM, Standaert TA, Williams-Warren J, Wadsworth SC, Smith AE, Welsh MJ (1996b) Repeat administration of an adenovirus vector encoding cystic fibrosis transmembrane conductance regulator to the nasal epithelium of patients with cystic fibrosis. J Clin Invest 97: 1504-1511.