ABCMdb

PMID: 12885347

Flotte TR, Zeitlin PL, Reynolds TC, Heald AE, Pedersen P, Beck S, Conrad CK, Brass-Ernst L, Humphries M, Sullivan K, Wetzel R, Taylor G, Carter BJ, Guggino WB
Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study.
Hum Gene Ther. 2003 Jul 20;14(11):1079-88., 2003-07-20 [PubMed]

Sentences

No. Mutations Sentence Comment

62 ABCC7 p.Gly551Asp Genotypes: homozygous or compound transfusion within 1 year or coughing of heterozygous DF508, G551D, . Login to comment 63 ABCC7 p.Trp1282* 30 ml more than once per week W1282X 4. Login to comment 145 ABCC7 p.Gly551Asp DEMOGRAPHICS AND BASELINE CHARACTERISTICS Low dose Medium High dose part 1 dose part 1 part 1 Part 2 (cohorts 1, (cohorts 4, (cohorts 7, (cohorts 10 2, and 3) 5, and 6) 8, and 9) and 11) Total Characteristic n 5 6 n 5 7 n 5 6 n 5 6 n 5 25 Age (years) 28.4 6 10.6 26.6 6 6.70 28.9 6 9.20 24.3 6 10.1 27.0 6 8.80 (range, 15 to 43) Height (cm) 168.0 6 11.40 171.1 6 13.30 171.5 6 10.70 157.5 6 44.80 170.4 6 11.00 Weight (kg) 60.5 6 9.90 71.2 6 19.9 63.7 6 8.80 66.8 6 11.6 65.8 6 13.4 Gender Male 3 (50.0) 5 (71.4) 4 (66.7) 5 (83.3) 17 (68.0) Female 3 (50.0) 2 (28.6) 2 (33.3) 1 (16.7) 08 (32.0) Race Caucasian 6 (100.0) 07 (100.0) 06 (100.0) 06 (100.0) 25 (100.0) CF Genotype DF508 homozygous 4 (66.7) 6 (85.7) 3 (50.0) 5 (83.3) 18 (72.0) DF508 heterozygous 2 (33.3) 1 (14.3) 2 (33.3) 1 (16.7) 06 (24.0) G551D heterozygous 0 (0.0)0 0 (0.0)0 1 (16.7) 0 (0.0)0 01 (4.0)0 Baseline PFT (Day 0) FEV1 (% predicted) 71.3 6 11.1 52.4 6 22.1 67.3 6 23.1 73.4 6 23.1 65.6 6 21.1 FVC (% predicted) 83.0 6 9.30 71.5 6 12.9 78.5 6 16.5 86.3 6 16. Login to comment