How does gene therapy work?

How does gene therapy work?

Gene therapy is intended to inject genetic material into cells to make up for abnormal genes or for generating a beneficial protein. In the case that a defective gene causes a damaged or absent protein, gene therapy may bring the gene into the normal copy to restore the protein function.

Typically a gene which is directly inserted into a cell does not operate. Rather, a carrier named a vector is designed to supply the gene. Some vectors are also used to deliver the new gene by infecting the cell. When used in humans, the viruses are changed to prevent disease.

In a chromosome in the human cell, some types of virus, such as retroviruses, contain their genetic material (including the new gene). Additional viruses, for example, adenoviruses, enter the DNA into the cell nucleus, but the DNA is not integrated into a chromosome.

A new gene is inserted directly into a cell. A carrier called a vector is genetically engineered to deliver the gene. An adenovirus introduces the DNA into the nucleus of the cell, but the DNA is not integrated into a chromosome.
A new gene is inserted directly into a cell. A carrier called a vector is genetically engineered to deliver the gene. An adenovirus introduces the DNA into the nucleus of the cell, but the DNA is not integrated into a chromosome.

The vector can directly be injected or injected into a certain tissue in a certain body (by IV) and is absorbed by each cell. A sample of the patient’s cells could otherwise be removed in a laboratory environment and exposed to the vector. The patient is then given back the cells holding the vector. The new gene supplied by the vector makes a working protein if treatment is successful.

Researchers have to solve several technical problems before the realistic solution to curing disease is gene therapy. For example, researchers need to find new ways of transmitting genes and targeting them for specific cells. They must also ensure that the body regulates new genes precisely.

How does gene therapy work?

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