What is in vivo gene therapy?

in vivo gene therapy is a process by which the genetic composition of cells is changed to create a therapeutic effect that prevents or treats diseases. A defective or missing hereditary material - DNA - in the nucleus of the patient's cells is changed or replaced by healthy genes. Specially modified viruses act as carriers of new genetic material and supply them to targeted cells or tissues of the patient. The genetic material is transferred in the patient's body during the in vivo gene therapy.

The process of the therapy in vivo is distinguished from the therapy by the ex vivo gene in that the last procedure takes cells from the patient's body, inserts genes and cultivates cells into the laboratory rather than inside the patient's body. Cells containing new DNA are later re -introduced into the necessary tissues in the patient. The Latin term ex vivo means "out of life" and in vivo means "in life". This treatment generally provides the extraction and replacement of the bone marrow of the patient in two separate operations.

carriers or vectors supplying therapeutic genetic material are retroviruses, adenoviruses, Herpes viruses and other viruses. The own genetic material of the viruses is removed or inactivated, which makes them harmless and unable to transmit the disease. The genetic material is then replaced by therapeutic genes. Various viruses focus on different cells and tissues in the body. They supply new genetic material in a similar way to a method used by viruses causing diseases when injuring genes into host cells in disease transmission.

Delivery

The gene is effective only if dividing cells are focused. In the therapeutic genes supplied during in vivo gene therapy to be expressed in the tissue, the cell divising process must be inserted into cells that pass through mitosis. This allows the new DNA to spread the patient's tissue. After the virus delivers new DNA, dies or is attacked by an immune system.

medical professionalThey also use gene therapy to treat various cancer, hereditary diseases caused by genetic defect, human immunodeficiency virus (HIV) and other diseases. This technique works best with diseases caused by individual defective genes. Many chronic diseases are caused by multiple gene disorders and represent a greater challenge.

Research on gene therapy in vivo focuses on improving specific vectors for targeted cells and tissues, which increases the efficiency of gene transfer, increasing the consistency of gene supply to the exact location in the patient's DNA sequence and exploring new applications to combat diseases for this technique. Research has also dealt with the problems of rejection of inserted therapeutic DNA and increase the long -term effectiveness of gene transfer. Effective, non -invasive methods of detection of gene expression in tissues and patient cells and cells after gene therapy in vivo have also explored.