Table 3

 Clinical trials

TrialGene expressionClinical efficacy
PVD, peripheral vascular disease; CAD, coronary artery disease; AV, adenoviral vector; AAV, adenoassociated viral vector; NGF, nerve growth factor; DMD, Duchenne muscular dystrophy; CF, cystic fibrosis; CFTR, cystic fibrosis transmembrane regulator.
CF
    Aerosolised AAV-CFTR phase I81Not detectedNot reported
    Maxillary sinus AAV-CFTR phase I82Not detectedNo improvement
    Aerosolised rAAV2-CFTR phase II83Limited expression with low copy number/cellAwaiting phase IIb results
DMD
    Phase I DMD naked plasmid DNA-dystrophin94,95Results pendingResults pending
Alzheimer’s disease
    Ex vivo NGF/fibroblasts phase I48Results pendingResults pending
Cancer
    P53 replacement AV-p53 recurrent glioma phase I145Limited to the injection siteModest survival benefit
    Phase II/III ovarian cancer AV-p53 replacement120Not determinedNo improvement
    Phase I non-small cell lung AV-p53 replacement116Not determinedSuggested survival benefit
    HSV-TK phase I mesothelioma125,126Not determinedNo improvement
    HSV-TK phase I refractotory brain cancer127–129Not determinedNo improvement
CAD/PVD
    BIOBYPASS phase I CAD139Not determinedIncreased exercise tolerance and increased myocardial perfusion
    BIOBYPASS phase I PVD135Not determinedIncreased exercise tolerance and increased ankle: brachial index. Improved quality of life