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| INVIVO TRANSFER OF THE HUMAN CYSTIC-FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR GENE TO THE AIRWAY EPITHELIUM |
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| Author(s): ROSENFELD MA, YOSHIMURA K, TRAPNELL BC, YONEYAMA K, ROSENTHAL ER, DALEMANS W, FUKAYAMA M, BARGON J, STIER LE, STRATFORDPERRICAUDET L, PERRICAUDET M, GUGGINO WB, PAVIRANI A, LECOCQ JP, CRYSTAL RG |
| Source: CELL Volume: 68 Issue: 1 Pages: 143-155 Published: JAN 10 1992 |
| Times Cited: 840 References: 53 |
| 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. |
| Document Type: Article |
| Language: English |
| Reprint Address: ROSENFELD, MA (reprint author), NHLBI, PULM BRANCH, BETHESDA, MD 20892 USA |
Addresses:
1. TRANSGENE SA, STRASBOURG, FRANCE
2. INST GUSTAVE ROUSSY, F-94805 VILLEJUIF, FRANCE
3. JOHNS HOPKINS UNIV, SCH MED, DEPT PHYSIOL, BALTIMORE, MD 21205 USA |
| Publisher: CELL PRESS, 1050 MASSACHUSETTES AVE, CIRCULATION DEPT, CAMBRIDGE, MA 02138 |
| Subject Category: Biochemistry & Molecular Biology; Cell Biology |
| IDS Number: GZ583 |
| ISSN: 0092-8674 |
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