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| CORRECTION OF THE ION-TRANSPORT DEFECT IN CYSTIC-FIBROSIS TRANSGENIC MICE BY GENE-THERAPY |
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| Author(s): HYDE SC, GILL DR, HIGGINS CF, TREZISE AEO, MACVINISH LJ, CUTHBERT AW, RATCLIFF R, EVANS MJ, COLLEDGE WH |
| Source: NATURE Volume: 362 Issue: 6417 Pages: 250-255 Published: MAR 18 1993 |
| Times Cited: 326 References: 33 |
| Abstract: CYSTIC fibrosis (CF) is a lethal inherited disorder affecting about 1 in 2,000 Caucasians. The major cause of morbidity is permanent lung damage resulting from ion transport abnormalities in airway epithelia that lead to mucus accumulation and bacterial colonization. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene1 that encodes a cyclic-AMP-regulated chloride channel2,3. Cyclic-AMP-regulated chloride conductances are altered in airway epithelia from CF patients4-6, suggesting that the functional expression of CFTR in the airways of CF patients may be a strategy for treatment. Transgenic mice7-9 With a disrupted cftr gene are appropriate for testing gene therapy protocols. Here we report the use of liposomes to deliver a CFTR expression plasmid to epithelia of the airway and to alveoli deep in the lung, leading to the correction of the ion conductance defects found in the trachea of transgenic (cf/cf) mice. These studies illustrate the feasibility of gene therapy for the pulmonary aspects of CF in humans. |
| Document Type: Article |
| Language: English |
Addresses:
1. UNIV OXFORD, JOHN RADCLIFFE HOSP, INST MOLEC MED, IMPERIAL CANC RES FUND LABS, OXFORD OX3 9DU, ENGLAND
2. UNIV CAMBRIDGE, DEPT PHARMACOL, CAMBRIDGE CB2 1QJ, ENGLAND
3. UNIV CAMBRIDGE, DEPT GENET, CAMBRIDGE CB2 1QR, ENGLAND
4. UNIV CAMBRIDGE, WELLCOME CRC INST CANC & DEV BIOL, CAMBRIDGE CB2 1QR, ENGLAND |
| Publisher: MACMILLAN MAGAZINES LTD, PORTERS SOUTH, 4 CRINAN ST, LONDON, ENGLAND N1 9XW |
| Subject Category: Multidisciplinary Sciences |
| IDS Number: KT026 |
| ISSN: 0028-0836 |
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