Gene therapy (also called human gene transfer) is a medical area that focuses on therapeutic delivery of nucleic acids into a patient's cells as a medicine to treat the disease. The first attempt to modify human DNA was made in 1980 by Martin Cline, but the first successful nuclear gene transfer in humans was approved by the National Institutes of Health in May 1983. The first direct insertion of human DNA into the DNA of the first therapeutic use was carried out by French Anderson in a trial in September 1990. It is time to think and treat them.

Between 1989 and December 2018, more than 2,900 clinical trials were conducted, more than half of them in phase I. In 2017, Spark Therapeutics from Luxturna (Blindness-induced mutation RPE65) and Kymriah from Novartis (therapy by chimeric antigen receptor) T cells) are the first FDA-approved gene therapies to enter the market. Since then, drugs such as Zolgensma from Novartis and Patisiran have been received by Alnylam from FDA approval, in addition to other gene therapy drugs from other companies. Most of these approvals use adeno-associated viruses (AAV) and lentiviruses to perform gene insertions, both in vivo and ex vivo. ASO / siRNA approaches such as those carried out by Alnylam and Ionis Pharmaceuticals require systems