Pharmacological gene therapy is a new field that combines pharmacological therapy and gene therapy. It is used either to prevent a defective gene from producing its protein or to increase the concentration of normal protein produced in the body by insertion of DNA or RNA fragments. It can also be using to generate immunity from contagious disease, such as TB, via the process of DNA vaccination.
Gene Therapeutics can be used to treat such conditions as Cystic Fibrosis (via the addition of a normal CFTR gene), Haemophilia A or even some of the complications of AIDS.
It uses a number of methods including: Gene augmentationTargeted Inhibition (using Antisense or antigene technology) Cell Killing (either direct or assisted) and DNA vaccination.