The potential for lentivector-mediated gene delivery to pancreatic cancer cells has not been systematically explored. Since their development almost a decade ago, 18 these vectors have undergone ...

Neurodegenerative diseases, typically characterized by a progressive nervous system dysfunction, represent a heavy burden both in terms of patient suffering and economic cost. The prevalence of ...

New viral vectors have been develoepd for efficiently delivering genes to neurons throughout the body and across the blood-brain barrier. Viruses have evolved to be highly effective vehicles for ...

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

Since the late 1990s, Charles Venditti, M.D., Ph.D., senior investigator with the National Human Genome Research Institute of the NIH, has been studying a group of rare and devastating pediatric ...

Moorfields Eye Hospital in London made international headlines last week. Doctors announced they had saved the sight of four young children suffering from a rare genetic condition that rapidly causes ...