Urea cycle disorders (UCDs) are inborn errors of ammonia detoxification/arginine synthesis
due to defects affecting the catalysts of the Krebs-Henseleit cycle (five core enzymes, one …

The urea cycle is a metabolic pathway for the disposal of excess nitrogen, which arises
primarily as ammonia. Nitrogen is essential for growth and life-maintenance, but excessive …

We report the death of an 18-year-old male with partial ornithine transcarbmaylase (OTC)
deficiency who participated in a pilot (safety) study of gene therapy. The vector used for this …

The first gene therapy clinical trials were initiated more than two decades ago. In the early
days, gene therapy shared the fate of many experimental medicine approaches and was …

The main objective in gene therapy is the development of efficient, non-toxic gene carriers
that can encapsulate and deliver foreign genetic materials into specific cell types such as …

▪ Abstract Broadly defined, the concept of gene therapy involves the transfer of genetic
material into a cell, tissue, or whole organ, with the goal of curing a disease or at least …

It has been 9 years since Mr. Jesse Gelsinger died from complications of vector
administration in a liver gene therapy trial of research subjects with a deficiency of ornithine …

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 …

Adenoviruses (Ad) are efficient vehicles for gene delivery in vitro and in vivo. Therefore, they
are a promising tool in gene therapy, particularly in the treatment of cancer and …

Recent conceptual and technical improvements have resulted in clinically meaningful levels
of gene transfer into repopulating hematopoietic stem cells. At the same time, evidence is …