Despite favorable DNA transfer efficiency, gene expression from recombinant adeno‐
associated virus (rAAV2) vectors in the lung has been variable in the context of cystic fibrosis …

The development of a successful gene therapy has many stages, including preclinical
testing in animal models and proof of principle clinical studies. A variety of diseases affect …

We previously reported that spliceosome-mediated RNA trans-splicing (SMaRT), using
recombinant adenoviral vectors expressing pre-trans-splicing molecules (PTMs), could …

The invention provides an isolated and purified DNA mol ecule comprising at least one DNA
segment, a biologically active Subunit or variant thereof, of a circular intermediate of adeno …

Gene and cell-based therapies are considered to be potentially powerful new approaches
for the management of cystic fibrosis (CF) lung disease. Despite tremendous efforts that …

Gene therapy using recombinant adeno-associated virus (rAAV2) vectors for cystic fibrosis
has shown gene transfer and remarkable safety, yet indeterminate expression. A new …

Recent advances in our understanding of the biology of the intervertebral disc have led to
increased interest in the development of novel treatments of intervertebral disc …

Numerous environmental influences have been demonstrated to enhance recombinant
adeno-associated virus (rAAV) transduction. Such findings are the foundation of developing …

Effective gene transfer with sustained gene expression is an important adjunct to the study of
intestinal inflammation and future therapy in inflammatory bowel disease. Recombinant …

Abstract Adeno-associated virus (AAV), a defective parvovirus, was discovered more than
30 years ago. Interest in this virus for human gene therapy applications focuses on its non …