How does gene therapy work?

Gene therapy means a technique of introducing a nucleic acid (DNA, RNA) into a patient’s cells for the purpose of treating certain diseases.

There are methods that use viral vectors (biological nano particles), i.e. modified viruses and non-viral methods based on DNA complexes (naked DNA).

When a virus infects an organism, it introduces its genetic material into the host cell and uses the mechanisms of that cell in order to produce proteins and replicate (multiply).

Viral vectors are tools used in molecular biology to transfer genetic material to a cell. Because diseases such as haemophilia involve a genetic defect, viral vectors are used to deliver non-modified copies of the genes to the patient’s body. One of the most secure alternatives in gene therapy is that of adenno-associated viruses (AAV – adeno-associated virus).

Non-viral methods are considered superior because they allow large-scale protein production and require a lower immune response from the host (any foreign element introduced into the organism involves a reaction from the immune system)


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