scholarly journals Functional Evidence of CFTR Gene Transfer in Nasal Epithelium of Cystic Fibrosis Mice in Vivo Following Luminal Application of DNA Complexes Targeted to the Serpin-Enzyme Complex Receptor

2002 ◽  
Vol 5 (4) ◽  
pp. 413-419 ◽  
Author(s):  
Assem-Galal Ziady ◽  
Thomas J. Kelley ◽  
Erin Milliken ◽  
Thomas Ferkol ◽  
Pamela B. Davis
Keyword(s):  
Cystic Fibrosis ◽  
Gene Transfer ◽  
Cftr Gene ◽  
Nasal Epithelium ◽  
Enzyme Complex ◽  
Dna Complexes ◽  
Complex Receptor

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

Gene Therapy ◽  
1997 ◽  
Vol 4 (3) ◽  
pp. 210-218 ◽  
Author(s):  
DJ Porteous ◽  
JR Dorin ◽  
G McLachlan ◽  
H Davidson-Smith ◽  
H Davidson ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Gene Transfer ◽  
Cationic Liposome ◽  
Cftr Gene ◽  
Nasal Epithelium ◽  
Safety And Efficacy

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

1995 ◽  
Vol 1 (1) ◽  
pp. 39-46 ◽  
Author(s):  
Natasha J. Caplen ◽  
Eric W.F.W. Alton ◽  
Peter G. Mddleton ◽  
Julia R. Dorin ◽  
Barbara J. Stevenson ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Gene Transfer ◽  
Cftr Gene ◽  
Nasal Epithelium

scholarly journals High lysosomal activities in cystic fibrosis tracheal gland cells corrected by adenovirus-mediated CFTR gene transfer

1999 ◽  
Vol 1453 (1) ◽  
pp. 14-22 ◽  
Author(s):  
W. Kammouni ◽  
D. Naı̈mi ◽  
W. Renaud ◽  
N. Bianco ◽  
C. Figarella ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Gene Transfer ◽  
Cftr Gene ◽  
Gland Cells

In vivo activation of CFTR-dependent chloride transport in murine airway epithelium by CNP

1997 ◽  
Vol 273 (5) ◽  
pp. L1065-L1072 ◽  
Author(s):  
Thomas J. Kelley ◽  
Calvin U. Cotton ◽  
Mitchell L. Drumm
Keyword(s):  
Cystic Fibrosis ◽  
Chloride Transport ◽  
Nasal Epithelium ◽  
Transmembrane Conductance Regulator ◽  
Wild Type ◽  
Transmembrane Conductance ◽  
Cyclic Monophosphate ◽  
Δf508 Cftr ◽  
Membrane Bound

Inhibitors of guanosine 3′,5′-cyclic monophosphate (cGMP)-inhibited phosphodiesterases stimulate Cl− transport across the nasal epithelia of cystic fibrosis mice carrying the ΔF508 mutation [cystic fibrosis transmembrane conductance regulator (CFTR) (ΔF/ΔF)], suggesting a role for cGMP in regulation of epithelial ion transport. Here we show that activation of membrane-bound guanylate cyclases by C-type natriuretic peptide (CNP) stimulates hyperpolarization of nasal epithelium in both wild-type and ΔF508 CFTR mice in vivo but not in nasal epithelium of mice lacking CFTR [CFTR(−/−)]. With the use of a nasal transepithelial potential difference (TEPD) assay, CNP was found to hyperpolarize lumen negative TEPD by 6.1 ± 0.6 mV in mice carrying wild-type CFTR. This value is consistent with that obtained with 8-bromoguanosine 3′,5′-cyclic monophosphate (6.2 ± 0.9 mV). A combination of the adenylate cyclase agonist forskolin and CNP demonstrated a synergistic ability to induce Cl− secretion across the nasal epithelium of CFTR(ΔF/ΔF) mice. No effect on TEPD was seen with this combination when used on CFTR(−/−) mice, implying that the CNP-induced change in TEPD in CFTR(ΔF/ΔF) mice is CFTR dependent.

scholarly journals CFTR gene transfer with AAV improves early cystic fibrosis pig phenotypes

JCI Insight ◽  
2016 ◽  
Vol 1 (14) ◽  
Author(s):  
Benjamin Steines ◽  
David D. Dickey ◽  
Jamie Bergen ◽  
Katherine J.D.A. Excoffon ◽  
John R. Weinstein ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Gene Transfer ◽  
Cftr Gene

scholarly journals Assessing the residual CFTR gene expression in human nasal epithelium cells bearing CFTR splicing mutations causing cystic fibrosis

2013 ◽  
Vol 22 (6) ◽  
pp. 784-791 ◽  
Author(s):  
Laia Masvidal ◽  
Susana Igreja ◽  
Maria D Ramos ◽  
Antoni Alvarez ◽  
Javier de Gracia ◽  
...  
Keyword(s):  
Gene Expression ◽  
Cystic Fibrosis ◽  
Cftr Gene ◽  
Nasal Epithelium ◽  
Human Nasal Epithelium ◽  
Splicing Mutations ◽  
Epithelium Cells

CFTR gene transfer to human cystic fibrosis pancreatic duct cells using a Sendai virus vector

2007 ◽  
Vol 214 (2) ◽  
pp. 442-455 ◽  
Author(s):  
Zoltán Rakonczay ◽  
Péter Hegyi ◽  
Mamoru Hasegawa ◽  
Makoto Inoue ◽  
Jun You ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Gene Transfer ◽  
Pancreatic Duct ◽  
Sendai Virus ◽  
Virus Vector ◽  
Cftr Gene ◽  
Duct Cells ◽  
Pancreatic Duct Cells

scholarly journals Safety and Biological Efficacy of a Lipid–CFTR Complex for Gene Transfer in the Nasal Epithelium of Adult Patients with Cystic Fibrosis

2000 ◽  
Vol 1 (1) ◽  
pp. 105-114 ◽  
Author(s):  
Peadar G. Noone ◽  
Katherine W. Hohneker ◽  
Zhaoqing Zhou ◽  
Larry G. Johnson ◽  
Carla Foy ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Gene Transfer ◽  
Adult Patients ◽  
Nasal Epithelium ◽  
Biological Efficacy
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