Gene therapy in the central nervous system (CNS) is hindered by the presence of the blood–brain barrier, which restricts access of serum constituents and peripheral cells to the brain parenchyma.

By Gina Kolata Researchers have corrected a disease-causing gene mutation with a single infusion carrying a treatment that precisely targeted the errant gene. This was the first time a mutated ...

delivering specific gene therapies directly to tumor sites. New data showed that a single dose of a tumor-selective triple knockout (“3KO”) RT virus with a specific immunotherapeutic payload ...