ADENO-ASSOCIATED VIRUS (AAV) VECTORS were first devel-oped 20 years ago (Carter,
2004) and an AAV vector was first administered to a human subject in November 1995 …

An ideal gene carrier requires both safety and transfection efficiency. Polyethylenimine (PEI)
is a well-known cationic polymer, which has high transfection efficiency owing to its buffering …

The airway is an important target for gene transfer to treat cystic fibrosis and other diseases
that affect the lung. We previously found that marker gene expression did not persist in the …

We sought to evaluate the ability of an E1–, E3–adenovirus (Ad) vector (AdGVCFTR. 10) to
transfer the normal human cystic fibrosis transmembrane conductance regulator (CFTR) …

To generate sufficient clinical-grade vector to support a phase I/II clinical trial of adeno-
associated virus serotype 8 (AAV8)-mediated factor IX (FIX) gene transfer for hemophilia B …

Gene therapy with adeno-associated viral (AAV) vectors has reached the clinical stage for
many inherited and acquired diseases. However, due to a cargo capacity limited to< 5 kb …

Adeno-associated virus (AAV) has emerged as an attractive vector for gene therapy. AAV
vectors have successfully been utilized to promote sustained gene expression in a variety of …

(AV. Luc) or the construct with a minimal promoter (AV. SV (P) Luc) alone gave only minimal
expression at 3 days after infection (Fig. 3a). However, co-infection with AV. SupEnh …

Clinical trials of gene therapy for cystic fibrosis suggest that current levels of gene transfer
efficiency are probably too low to result in clinical benefit, largely as a result of the barriers …

Recombinant AAV efficacy has been demonstrated in numerous gene therapy preclinical
studies. As this vector is increasingly applied to human clinical trials, it is a priority to …