A plenty of diseases which cannot be properly cured by the current healthcare tools and drugs pushes scientists to search for alternative ways of treatment. Gene therapy is one of the experimental techniques which promises to get rid of incurable diseases once and for all. Gene therapy is an insertion of a life-saving gene into the patient’s body or replacement of the mutated one with a healthy copy. The technique is claimed to be successful in curing various congenital diseases, some types of cancer, and rare conditions which result from the mutation of genes.
The mechanism of genetic substitution seems to be quite clear. The new genetic material in the therapy is usually introduced to the cell with the help of a carrier called a vector. Viruses are frequently used as vectors because they easily infect a cell and change its genetic structure. There are retroviruses which integrate their genetic material into a human chromosome and adenoviruses which deliver their DNA to the nucleus of the human cell. If the manipulation is successful, the human cell makes functional protein using a new gene. Viruses used as vectors are modified in the laboratory so that they cannot infect the patients.
Gene therapy was first tested in clinical trials in 1990. Revolutionary technique promised miraculous recovery, but the first death from an experimental gene therapy came in 1999, and the craze on the issue died down. Nevertheless, today clinical trials successfully continue, and doctors have managed to cure the patients of hemophilia and rare inherited blindness. Presumably, Spark Therapeutics’ treatment for blindness will become the first type of gene therapy legally approved in the US.