La thérapie génique c’est quoi ?
Understanding Gene Therapy
The Role of DNA in Organ Function
- Cells that make up our organs contain genetic material (DNA) specific to each individual, which is crucial for the production of proteins.
- DNA consists of an alphabet made up of four letters: A, T, C, and G. Mutations or errors in this genetic alphabet can lead to dysfunctional proteins and diseases.
Introduction to Gene Therapy
- Gene therapy involves introducing a therapeutic gene into cells to ensure proper protein synthesis. This method leverages the natural ability of viruses to deliver genetic material into target cells.
- Scientists modify viruses by removing their pathogenic characteristics and adding therapeutic genes, transforming them into effective delivery vehicles for treatment.
Types of Viral Vectors Used
- There are four types of viral vectors used in gene therapy; two notable ones studied at Sanofi include AVP (Adeno-associated virus) and lentivirus.
- AVPs specifically target diseased cells by delivering therapeutic genes directly into the cell nucleus, while lentiviruses convert their RNA into DNA before integrating it into the host's genome.
Mechanism and Impact on Disease Treatment
- Once inside the nucleus, the introduced therapeutic gene enables cells to produce functional proteins, potentially curing diseases such as cancer or rare genetic disorders.