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| NONINVASIVE LIPOSOME-MEDIATED GENE DELIVERY CAN CORRECT THE ION-TRANSPORT DEFECT IN CYSTIC-FIBROSIS MUTANT MICE |
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| Author(s): ALTON EWFW, MIDDLETON PG, CAPLEN NJ, SMITH SN, STEEL DM, MUNKONGE FM, JEFFERY PK, GEDDES DM, HART SL, WILLIAMSON R, FASOLD KI, MILLER AD, DICKINSON P, STEVENSON BJ, MCLACHLAN G, DORIN JR, PORTEOUS DJ |
| Source: NATURE GENETICS Volume: 5 Issue: 2 Pages: 135-142 Published: OCT 1993 |
| Times Cited: 332 References: 33 |
| Abstract: We report gene transfer to the Edinburgh insertional mutant mouse (cf/cf), delivering CFTR cDNA-liposome complexes into the airways by nebulization. We show full restoration of cAMP related chloride responses in some animals and demonstrate, in the same tissues, human CFTR cDNA expression. Overall, a range of correction was seen with restoration of about 50% of the deficit between wild type mice and untreated cf/cf controls. We report modest correction in the intestinal tract following direct instillation and provide initial encouraging safety data for both the respiratory and intestinal tract following the liposome mediated gene delivery. The non-viral nature and potentially lower immunogenicity of DNA-liposomes suggest that this may offer a therapeutic alternative to adenoviral therapies. |
| Document Type: Article |
| Language: English |
| Reprint Address: ALTON, EWFW (reprint author), NATL HEART & LUNG INST, ION TRANSPORT LAB, LONDON SW3 6LR, ENGLAND |
Addresses:
1. NATL HEART & LUNG INST, DEPT LUNG PATHOL, LONDON SW3 6LR, ENGLAND
2. ST MARYS HOSP, SCH MED, DEPT BIOCHEM & MOLEC GENET, LONDON W2 1PG, ENGLAND
3. UNIV LONDON IMPERIAL COLL SCI & TECHNOL, DEPT CHEM, LONDON SW7 1NA, ENGLAND
4. WESTERN GEN HOSP, MRC, HUMAN GENET UNIT, EDINBURGH EH4 2XU, MIDLOTHIAN SCOTLAND |
| Publisher: NATURE PUBLISHING CO, 345 PARK AVE SOUTH, NEW YORK, NY 10010-1707 |
| Subject Category: Genetics & Heredity |
| IDS Number: MA056 |
| ISSN: 1061-4036 |
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