New trends in a human gene therapy
According to U.S. Food and Drug Administration (FDA), gene therapy is "an experimental technique that modifies a person's genes to treat or cure disease by replacing disease-causing genes with healthy engineered gene constructs, inactivating malfunctioning genes, or introducing new genes into the body to treat a disease". A recent article in Biomedicine & Pharmacotherapy Journal provides an overview on the latest technology advances. For example, CRISPR-Cas9 is a promising gene editing system that allows the treatment of such genetic pathologies as retinal diseases, HIV, and liver diseases. Another revolutionary new technology is CAR-based immunotherapy that is aimed to treat leukemias and lymphomas. The principle is that it helps to direct genetically modified T lymphocytes toward tumor cells. Furthermore, efficient gene delivery to desired cells and tissues can be achieved with nanomedicine, avoiding a potential immune response to viral vectors. Authors state that such therapeutic techniques are much more efficient that conventional drugs. Nonetheless, safety of gene therapy is still the main issue, along with insufficient technology level, affordability and lack of regulations.