Introduction to Gene Therapy


Gene therapy is a technique that involves inserting genetic material into cells in order to compensate for abnormal genes or produce a beneficial protein. If a defective gene causes a required protein to be faulty or absent, gene therapy may be able to restore the protein's function by introducing a regular copy of the gene. In most cases, a gene that is inserted directly into a cell does not function. Instead, the gene is delivered by a genetically modified carrier called a vector. Since they can deliver the new gene by infecting the cell, such viruses are often used as vectors. When the viruses are used in humans, they are changed so that they do not cause disease. Retroviruses, for example, insert their genetic material (which includes the new gene) into a chromosome in the human cell. Adenoviruses, for example, insert their DNA into the cell's nucleus, but the DNA is not integrated into a chromosome. Gene therapy is being tested in clinical trials for cancer, age-related macular degeneration, and other eye disorders, as well as some genetic conditions and HIV/AIDS. The US Food and Drug Administration (FDA) has approved one gene therapy treatment, Luxturna®, for use in the United States. Luxturna is a drug that is used to treat hereditary retinal (eye) disorders. Doctors carry a safe copy of a gene to cells within the body using gene therapy. This healthy gene can be used to replace a defective (mutated) gene, inactivate a mutated gene, or even add a new gene. Vectors, or carriers, transport healthy genes into cells. The vectors are usually modified viruses that do not cause disease. Certain bacteria or circular DNA molecules may also serve as vectors (plasmid DNA). Additional approaches for packaging and delivering genetic content, such as the use of nanoparticles, encapsulating lipid molecules, and using electric currents, are also being studied. Vectors are introduced into the body by injection or intravenous (IV) infusion. In certain cases, doctors extract cells from a patient, add vectors in a laboratory, and then inject or IV infuse the vector-containing cells back into the patient's body. Those who are interested to publish their article in our journal, they can submit it either send it as an email attachment to this below given mail id or submit it online through given link:

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Journal of Medical and Surgical Pathology

ISSN: 2472-4971 | NLM ID: 101245791