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Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis.

Caplen NJ, Alton EW, Middleton PG, Dorin JR, Stevenson BJ, Gao X, Durham SR, Jeffery PK, Hodson ME, Coutelle C, et al.

Nat Med. 1995 Jan;1(1):39-46. Erratum in: Nat Med 1995 Mar;1(3):272.


Safety and biological efficacy of a lipid-CFTR complex for gene transfer in the nasal epithelium of adult patients with cystic fibrosis.

Noone PG, Hohneker KW, Zhou Z, Johnson LG, Foy C, Gipson C, Jones K, Noah TL, Leigh MW, Schwartzbach C, Efthimiou J, Pearlman R, Boucher RC, Knowles MR.

Mol Ther. 2000 Jan;1(1):105-14.


A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis.

Gill DR, Southern KW, Mofford KA, Seddon T, Huang L, Sorgi F, Thomson A, MacVinish LJ, Ratcliff R, Bilton D, Lane DJ, Littlewood JM, Webb AK, Middleton PG, Colledge WH, Cuthbert AW, Evans MJ, Higgins CF, Hyde SC.

Gene Ther. 1997 Mar;4(3):199-209.


Evidence for safety and efficacy of DOTAP cationic liposome mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis.

Porteous DJ, Dorin JR, McLachlan G, Davidson-Smith H, Davidson H, Stevenson BJ, Carothers AD, Wallace WA, Moralee S, Hoenes C, Kallmeyer G, Michaelis U, Naujoks K, Ho LP, Samways JM, Imrie M, Greening AP, Innes JA.

Gene Ther. 1997 Mar;4(3):210-8.


Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis.

Hyde SC, Southern KW, Gileadi U, Fitzjohn EM, Mofford KA, Waddell BE, Gooi HC, Goddard CA, Hannavy K, Smyth SE, Egan JJ, Sorgi FL, Huang L, Cuthbert AW, Evans MJ, Colledge WH, Higgins CF, Webb AK, Gill DR.

Gene Ther. 2000 Jul;7(13):1156-65.


A double-blind, placebo controlled, dose ranging study to evaluate the safety and biological efficacy of the lipid-DNA complex GR213487B in the nasal epithelium of adult patients with cystic fibrosis.

Knowles MR, Noone PG, Hohneker K, Johnson LG, Boucher RC, Efthimiou J, Crawford C, Brown R, Schwartzbach C, Pearlman R.

Hum Gene Ther. 1998 Jan 20;9(2):249-69.


A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis.

Knowles MR, Hohneker KW, Zhou Z, Olsen JC, Noah TL, Hu PC, Leigh MW, Engelhardt JF, Edwards LJ, Jones KR, et al.

N Engl J Med. 1995 Sep 28;333(13):823-31.


Vector-specific complementation profiles of two independent primary defects in cystic fibrosis airways.

Zhang Y, Jiang Q, Dudus L, Yankaskas JR, Engelhardt JF.

Hum Gene Ther. 1998 Mar 20;9(5):635-48.


Increased contact time improves adenovirus-mediated CFTR gene transfer to nasal epithelium of CF mice.

Jiang C, Akita GY, Colledge WH, Ratcliff RA, Evans MJ, Hehir KM, St George JA, Wadsworth SC, Cheng SH.

Hum Gene Ther. 1997 Apr 10;8(6):671-80.


Optimisation of real-time quantitative RT-PCR for the evaluation of non-viral mediated gene transfer to the airways.

Rose AC, Goddard CA, Colledge WH, Cheng SH, Gill DR, Hyde SC.

Gene Ther. 2002 Oct;9(19):1312-20.


Efficiency of cationic lipid-mediated transfection of polarized and differentiated airway epithelial cells in vitro and in vivo.

Jiang C, O'Connor SP, Fang SL, Wang KX, Marshall J, Williams JL, Wilburn B, Echelard Y, Cheng SH.

Hum Gene Ther. 1998 Jul 20;9(11):1531-42.


Laboratory and clinical studies in support of cystic fibrosis gene therapy using pCMV-CFTR-DOTAP.

McLachlan G, Ho LP, Davidson-Smith H, Samways J, Davidson H, Stevenson BJ, Carothers AD, Alton EW, Middleton PG, Smith SN, Kallmeyer G, Michaelis U, Seeber S, Naujoks K, Greening AP, Innes JA, Dorin JR, Porteous DJ.

Gene Ther. 1996 Dec;3(12):1113-23.


Compacted DNA nanoparticles administered to the nasal mucosa of cystic fibrosis subjects are safe and demonstrate partial to complete cystic fibrosis transmembrane regulator reconstitution.

Konstan MW, Davis PB, Wagener JS, Hilliard KA, Stern RC, Milgram LJ, Kowalczyk TH, Hyatt SL, Fink TL, Gedeon CR, Oette SM, Payne JM, Muhammad O, Ziady AG, Moen RC, Cooper MJ.

Hum Gene Ther. 2004 Dec;15(12):1255-69.


Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus.

Wagner JA, Messner AH, Moran ML, Daifuku R, Kouyama K, Desch JK, Manley S, Norbash AM, Conrad CK, Friborg S, Reynolds T, Guggino WB, Moss RB, Carter BJ, Wine JJ, Flotte TR, Gardner P.

Laryngoscope. 1999 Feb;109(2 Pt 1):266-74.


A second dose of a CFTR cDNA-liposome complex is as effective as the first dose in restoring cAMP-dependent chloride secretion to null CF mice trachea.

Goddard CA, Ratcliff R, Anderson JR, Glenn E, Brown S, Gill DR, Hyde SC, MacVinish LJ, Huang L, Higgins CF, Cuthbert AW, Evans MJ, Colledge WH.

Gene Ther. 1997 Nov;4(11):1231-6.


Aerosol and lobar administration of a recombinant adenovirus to individuals with cystic fibrosis. I. Methods, safety, and clinical implications.

Joseph PM, O'Sullivan BP, Lapey A, Dorkin H, Oren J, Balfour R, Perricone MA, Rosenberg M, Wadsworth SC, Smith AE, St George JA, Meeker DP.

Hum Gene Ther. 2001 Jul 20;12(11):1369-82.


Expression of CFTR from a ciliated cell-specific promoter is ineffective at correcting nasal potential difference in CF mice.

Ostrowski LE, Yin W, Diggs PS, Rogers TD, O'Neal WK, Grubb BR.

Gene Ther. 2007 Oct;14(20):1492-501. Epub 2007 Jul 19.


Cationic lipids for reporter gene and CFTR transfer to rat pulmonary epithelium.

Logan JJ, Bebok Z, Walker LC, Peng S, Felgner PL, Siegal GP, Frizzell RA, Dong J, Howard M, Matalon, et al.

Gene Ther. 1995 Jan;2(1):38-49.


CFTR transgene expression in primary DeltaF508 epithelial cell cultures from human nasal polyps following gene transfer with cationic phosphonolipids.

Montier T, Delépine P, Marianowski R, Le Ny K, Le Bris M, Gillet D, Potard G, Mondine P, Frachon I, Yaouanc JJ, Clément JC, Des Abbayes H, Férec C.

Mol Biotechnol. 2004 Mar;26(3):193-206.

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