Gene therapy medicinal product is a biological medicinal product. This product aims to regulate, repair, replace, add or delete a genetic sequence. Gene transfer vectors which are now producing clinical results. In September 14th 1990 the FDA approved the first time a gene therapy trial with a therapeutic attempt in humans. Two children suffering from adenosine deaminase deficiency (ADA-SCID). From the data was published in 2013, more than 1700 gene therapy medicinal products approved clinical trials (Thomas et al., 2013).
For the in vivo application of gene-based drugs, the therapeutic gene is introduced directly into the body (e.g. muscle, liver) of the patient, while for ex vivo applications, patient cells are first isolated from the body, genetically modified outside the body and reintroduced into the patient as an autologous transplant (Kerstin et al., 2013)
The knowledge gained within the field over the past several decades provides much hope for the future of gene therapy. The success of gene therapy has largely been driven by improvements in non-viral and viral gene transfer vectors. An array of physical and chemical non-viral methods have been used to transfer DNA and mRNA (Keeler et al., 2017).
From the review of Kerstin et al., (2013), the risks associated with gene therapy are already being successfully addressed.
REFERENCES
Keeler, ElMallah and Flotte, 2017. Gene Therapy 2017: Progress and Future Directions, Clin Transl Sci 10: 242–248. Doi:10.1111/cts.12466.
. Kerstin B. Kaufmann, Hildegard Buning, Anne Galy, Axel Schambach, Manuel Grez, 2013. Gene therapy on the move, EMBO Mol Med, 5: 1642–1661. Doi: 10.1002/emmm.201202287.
. Thomas Wirth, Nigel Parker, Seppo Ylä-Herttuala, 2013. History of gene therapy, Gene, 525: 162–169.