Gene therapy is a revolutionary treatment designed to introduce a functional copy of a gene into cells in place of missing or defective ones in order to correct genetic disorders. Therapeutic effect can be achieved through insertion, deletion, inactivation, or modification of genetic sequences. 

RNA therapeutics are a new class of medications based on ribonucleic acid (RNA). These drugs can either trigger protein synthesis to promote a new therapeutics effect, such as a vaccine, or they can activate an interfering mechanism in order to suppress pathological protein pathway expression and block the disease. 

Cell therapy is a therapy in which viable cells are injected or implanted into a patient to trigger a medicinal effect. These cells are originally collated from a patient, cultured and transformed in vitro with therapeutic genetic material. The transformed cells which have the capability to cure the symptoms are re-injected in the patient.