ABCMdb

PMID: 11139834

Shah PL
Update on clinical trials in the treatment of pulmonary disease in patients with cystic fibrosis.
Expert Opin Investig Drugs. 1999 Nov;8(11):1917-1927., [PubMed]

Sentences

No. Mutations Sentence Comment

43 ABCC7 p.Gly542* A pilot study has thus far treated eight patients with the G542X mutation with iv. Login to comment 57 ABCC7 p.Gly551Asp ABCC7 p.Trp1282* ABCC7 p.Gly542* (Genetech, Roche, NIH) & University of Cornell Repeated dosing, every 30 days to the Lungs Age > 15 years FEV1 > 40% predicted 26 Degree of transfection, duration and stability of expression Adenovirus vector Phase II University of Iowa Repeated administration to the lung NK NK Degree of transfection, duration and stability of expression Adeno-associated viral vector Phase I Targeted Genetics, Medeva, Stanford University, University of Washington & Boston Children Hospital Aerosol to lung Age > 15 years FVC > 40% predicted CF genotypes: DF508, G551D, W1282X, and G542X 12 Degree of transfection Cationic lipids, Cytofectins Phase I Genzyme, Vical, University of Alabama Lung Age > 18 years FEV1 > 40% predicted NK Degree of transfection Liposomal DNA complexes Phase II NHLI, UK Inhalation to lung Male FEV1 > 70% 16 i) functional correction ii) degree of transfection iii) bacterial adherence NHLI: National Heart & Lung Institute (Imperial College, UK); NIH: National Institute of Health (USA); NK: Not known. Login to comment