A phase 1 study, in cystic fibrosis patients, of the safety, toxicity, and biological efficacy of a single administration of a replication deficient, recombinant adenovirus carrying the cDNA of the normal cystic fibrosis transmembrane conductance regulator gene in the lung.
Crystal
R G
RG
Rockefeller University Hospital, New York, New York 10021, USA.
Jaffe
A
A
Brody
S
S
Mastrangeli
A
A
McElvaney
N G
NG
Rosenfeld
M
M
Chu
C S
CS
Danel
C
C
Hay
J
J
Eissa
T
T
D000256:Adenoviridae / Q000235:genetics*; D000328:Adult; D001980:Bronchi / Q000378:metabolism; D002985:Clinical Protocols; D017321:Clinical Trials, Phase I as Topic; D003550:Cystic Fibrosis / Q000235:genetics / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000096:biosynthesis / Q000235:genetics*; D018076:DNA, Complementary / Q000235:genetics; D004848:Epithelium / Q000378:metabolism; D005260:Female; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000009:adverse effects; D005822:Genetic Vectors; D006801:Humans; D008297:Male; D009297:Nasal Mucosa / Q000378:metabolism; D018579:Patient Selection; D011994:Recombinant Proteins / Q000235:genetics; D014779:Virus Replication
C505032:CFTR protein, human; D018076:DNA, Complementary; D011994:Recombinant Proteins; D019005:Cystic Fibrosis Transmembrane Conductance Regulator