Therapeutics

In medical research, Genome engineering is the basis of gene therapy. For example, it can enable a defective gene to be replaced by its functional copy.This type of therapy, often known as “genome surgery”, could be used to cure a large proportion of genetic diseases.

Cellectis is particularly involved in research into falciform anemia (or sickle-cell disease), a genetic disease affecting the molecule that transports oxygen in the blood, hemoglobin. If a healthy gene is inserted into the cells of the organs that produce red blood cells, the patient’s body will start producing healthy red blood cells again. Inserm, the CNRS and AFM are closely involved in Cellectis’ progress in this field.

Moreover, Genome engineering can also be used to fight viruses such as HIV. These pathogens insert their DNA into the DNA of infected cells, meaning that the immune system is unable to rid itself of them permanently. Cellectis’ ability to target very precise regions of the Genome and inactivate them may make it possible to remove the genetic material of the viruses found in the infected cells; this therefore represents a potential breakthrough in the treatment of these diseases.

In brief, Cellectis’ technology allows the replacement of a defective gene by a functional gene (monogenic dis¬eases such as muscular dystrophy or hemophilia), the suppression of persistent viral DNA (HIV, hepatitis B or herpes viruses) and the creation of Stem cell lines with particular characteristics. Stem cells have indeed a strong potential when it comes to therapy and industry. They enable to generate cells, and therefore tissues, of any type and with any kind of genome.