The hereditary form comprises≈ 1/5 of patients with dilated cardiomyopathy (DCM) and is a
major cause of advanced heart failure. Medical and socioeconomic settings require novel …

Despite extensive experience with recombinant adeno-associated virus (rAAV) 2 vectors in
the lung, gene expression has been low in the context of cystic fibrosis (CF) gene therapy …

Study Design The design of the initial clinical studies of AAV-mediated gene transfer for
hemophilia B was influenced by the very limited clinical experience with AAV vectors at that …

Cystic fibrosis (CF) is an autosomal recessive disease that is potentially treatable by gene
therapy. Since the identification of the gene encoding CF transmembrane conductance …

Despite significant advances in cystic fibrosis (CF) treatments, a one-time treatment for this
life-shortening disease remains elusive. Stable complementation of the disease-causing …

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 …

Gene therapy is an alternative therapy in many respiratory diseases with genetic origin and
currently without curative treatment. After five decades of progress, many different vectors …

Background Chronic rhinosinusitis frequently occurs in people with cystic fibrosis. Several
medical interventions are available for treating chronic rhinosinusitis in people with cystic …

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

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 …