Cancer is one of the leading causes of death in the United States, claiming over half a million lives each year. While doctors and researchers understand the basic causes of cancer, they are still developing methods to effectively treat this deadly disease. One new type of cancer treatment that is currently in clinical trials is called gene therapy.

Female induced pluripotent stem (iPS) cells, reprogrammed from human skin cells into cells that have the embryonic-like potential to become any cell in the body, retain an inactive X chromosome, stem cell researchers at UCLA have found. The finding could have implications for studying X chromosome-linked diseases such as Rett syndrome, caused by mutations in a gene located on the X chromosome.

Polymorphisms in the IL28B (interleukin-28B) gene region are important in predicting outcome following therapy for chronic hepatitis C virus (HCV) infection. We evaluated the role of IL28B in spontaneous and treatment-induced clearance following recent HCV infection.

Leptomycin B (LMB) and/or its derivatives are considered a novel class of cancer therapeutics through blocking chromosome maintenance region 1, which mediates p53 nuclear export. The objectives of the present study were to first evaluate the cytotoxic effects of LMB on a normal human lung epithelial cell line (BEAS-2B) and three human lung adenocarcinoma cell lines with various p53 status (wild type: A549, mutant: NCI-H522, and null: NCI-H358) and then to identify LMB-induced gene expression alterations in human p53 signaling pathway.

Virus-like particles (VLPs) of influenza virus have been proposed as an equally safe and efficacious alternative to the conventional reverse-genetic vaccine platform. VLPs can be produced in a variety of cell culture systems including insect cell lines, which typically entail use of the Baculovirus Expression Vector System (BEVS) ...

Impaired wound healing in diabetes is related to decreased production of growth factors. Hence, gene therapy is considered as promising treatment modality. So far, efforts concentrated on single gene therapy with particular emphasis on vascular endothelial growth factor-A (VEGF-A). However, as multiple proteins are involved in this process it is rational to test new approaches. Therefore, the aim of this study was to investigate whether single AAV vector-mediated transfer of VEGF-A and fibroblast growth factor 4 (FGF4) coding sequences will improve the wound healing over the effect of VEGF-A in diabetic (db/db) mice.

Arming a replicating adenovirus with osteoprotegerin reduces the tumor burden in a murine model of osteolytic bone metastases of breast cancer.

Most patients with advanced breast cancer develop osteolytic bone metastases, which have numerous complications. Because current therapies are not curative, new treatments are needed. Conditionally replicating adenoviruses (CRAds) are anticancer agents designed to infect and lyse tumor cells.

CanScript, an 18-Base pair DNA sequence, boosts tumor cell-specific promoter activity implications for targeted gene therapy.

Gene therapy protocols for the treatment of cancer often employ gene promoter sequences that are known to be overexpressed in specific tumor cell types relative to normal cells. These promoters, while specific, are often weakly active. It would be desirable to increase the activity of such promoters, while at the same time retain specificity, so that the therapeutic gene is more robustly expressed.

After more than a decade of research, Scripps Research Institute scientists have pieced together the structure of a human adenovirus—the largest complex ever determined at atomic resolution. The new findings about the virus, which causes respiratory, eye, and gastrointestinal infections, may lead to more effective gene therapy and to new anti-viral drugs.

Researchers in Lund Sweden led by Professor Merab Kokaia, are first to introduce genes into the brain that induce production of specific neuropeptide receptors that when stimulated by neuropeptide Y (NPY) can suppress epilepsy in experimental animals, as reported in the journal Brain. Since NPY can stimulate many brain receptors, some of which can increase the risk of seizures, increasing the inhibitory receptor population may provide therapeutic advantages.

Gene targeting drug therapy may offer hope for those with difficult-to-treat advanced-stage skin cancer according to preliminary data published Wednesday in the New England Journal of Medicine.