Gene therapy has a history of controversy. Encouraging results are starting to emerge from
the clinic, but questions are still being asked about the safety of this new molecular …

Circumventing the immune response to the vector is a major challenge with all vector types.
Viral vectors are the most likely to induce an immune response, especially those, like …

The concept behind gene therapy is simple—by delivering corrective genetic material to the
cells of a patient the symptoms of disease can be alleviated. But seven years after the first …

The art and science of gene therapy has received much attention of late. The tragic death of
18-year-old Jesse Gelsinger, a volunteer in a Phase I clinical trial, has overshadowed the …

Chronic liver diseases are frequent and potentially life threatening for humans. The
underlying etiologies are diverse, ranging from viral infections, autoimmune disorders, and …

Inherited retinal dystrophies (IRDs) are a group of clinically and genetically heterogeneous
degenerative disorders. To date, mutations have been associated with IRDs in over 270 …

Gene therapy is a clinical strategy that may potentially treat an array of genetic and
nongenetic diseases, as well as a novel method for drug delivery and vaccination. To these …

Gene therapy can be broadly defined as the transfer of genetic material to cure a disease or
at least to improve the clinical status of a patient. One of the basic concepts of gene therapy …

Abstract The Sleeping Beauty (SB) transposon system, derived from teleost fish sequences,
is extremely effective at delivering DNA to vertebrate genomes, including those of humans …

Recent endeavors in the development of adenovirus as a gene vector have focused on the
modification of virus tropism, the accomodation of larger genes, and the increase in stability …