@article{dbfcd14aaf0943fdb8b0fd54e6de2b6d,
title = "In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium",
abstract = "Direct transfer of the normal cystic fibrosis (CF) transmembrane conductance regulator (CFTR) gene to airway epithelium was evaluated using a replication-deficient recombinant adenovirus (Ad) vector containing normal human CFTR cDNA (Ad-CFTR). In vitro Ad-CFTR-infected CFPAC-1 CF epithelial cells expressed human CFTR mRNA and protein and demonstrated correction of defective cAMP-mediated Cl- permeability. Two days after in vivo intratracheal introduction of Ad-CFTR in cotton rats, in situ analysis demonstrated human CFTR gene expression in lung epithelium. PCR amplification of reverse transcribed lung RNA demonstrated human CFTR transcripts derived from Ad-CFTR, and Northern analysis of lung RNA revealed human CFTR transcripts for up to 6 weeks. Human CFTR protein was detected in epithelial cells using anti-human CFTR antibody 11-14 days after infection. While the safety and effectiveness remain to be demonstrated, these observations suggest the feasibility of in vivo CFTR gene transfer as therapy for the pulmonary manifestations of CF.",
author = "Rosenfeld, {Melissa A.} and Kunihiko Yoshimura and Trapnell, {Bruce C.} and Koichi Yoneyama and Rosenthal, {Eugene R.} and Wilfried Dalemans and Masashi Fukayama and Joachim Bargon and Stier, {Larue E.} and Leslie Stratford-Perricaudet and Michel Perricaudet and Guggino, {William B.} and Andrea Pavirani and Lecocq, {Jean Pierre} and Crystal, {Ronald G.}",
note = "Funding Information: We thank R. Friuell (Department of Physiology, University of Alabama) for the generous gift of CFPAC-1 cells; T. Banks and D. Dreyer for technical assistance; S. Rosenfeld (Laboratory of Clinical Hematology, National Heart, Lung and Blood Institute), C. Danel. and R. Wersto for assistance with immunohistochemistry; D. Villeval for sequence analysis; R. Dreyfuss for help with photomicroscopy; G. Prince and M. Reddington (Laboratory of Infectious Diseases, National Institute of Allergy and Infectious Diseases) for making cotton rats available to us; T. Shenkfor thegiftofAd-dl312;andT. Raymerforeditorialassistance. This work was supported in part by the American Cystic Fibrosis Foundation and I{\textquoteright}Association Francaise de lutte contre la Mucoviscidose. W. G. was supported by the Cystic Fibrosis Foundation Research Development Program and NIH grants HL 40176 and HL 47122. The costs of publication of this article were defrayed in part by the payment of page charges. This article must therefore be hereby marked “advetiisement” in accordance with 16 USC Section 1734 solely to indicate this fact.",
year = "1992",
month = jan,
day = "10",
doi = "10.1016/0092-8674(92)90213-V",
language = "English (US)",
volume = "68",
pages = "143--155",
journal = "Cell",
issn = "0092-8674",
publisher = "Cell Press",
number = "1",
}