Clinical trials in cystic fibrosis (CF) patients established proof-of-principle for transfer of the
wild-type cystic fibrosis transmembrane conductance regulator (CFTR) gene to airway …

Recent advances in high-throughput molecular testing have made it possible to diagnose
most genetic disorders relatively early in gestation with minimal risk to the fetus. These …

Adeno-associated virus vectors (AAV) show promise for liver-targeted gene therapy. In this
study, we examined the long-term consequences of a single intravenous administration of a …

Transduction with recombinant adeno-associated virus (AAV) vectors is limited by the need
to convert its single-stranded (ss) genome to transcriptionally active double-stranded (ds) …

Amutation in the small GTPase Rab38 gives rise to the mouse coat color phenotype
“chocolate”(cht), implicating Rab38 in the regulation of melanogenesis. However, its role …

Upon local delivery, adenovirus (Ad) serotype 5 viruses use the coxsackie and Ad receptor
(CAR) for cell binding and αv integrins for internalization. When administered systemically …

Gene therapy by use of integrating vectors carrying therapeutic transgene sequences offers
the potential for a permanent cure of genetic diseases by stable vector insertion into the …

Several diseases of the nervous system are characterized by neurodegeneration and death
in childhood. Conventional medicine is ineffective, but fetal or neonatal gene therapy may …

The major complication associated with protein replacement therapy currently used in the
treatment of hemophilia B (HB) is the development of antibodies to the infused human Factor …

Hemophilia is an X-linked inherited bleeding disorder consisting of two classifications,
hemophilia A and hemophilia B, depending on the underlying mutation. Although the …