Gene Therapy and Its Importance

In this gene therapy and its importance post we have briefly explained about gene therapy process, current approaches, Somatic cell gene therapy process, and Germ cell gene therapy process.

Gene Therapy Process

Biochemistry and molecular biology advancements have aided in the understanding of the genetic basis of inherited disorders. The researchers objective was to replace defective genes with good ones and treat genetic diseases.

The method of putting genes into cells to treat diseases is known as gene therapy process.  The newly discovered genes will code for proteins and rectify inadequacies in hereditary  disorders.  As a result, gene therapy process typically entails genetic alterations in animals or people to treat disease and maintain health. Experiments are first carried out on animals, then on humans. Clearly, the researchers purpose is to serve humanity and improve their health.

In gene augmentation therapy, a DNA is inserted into the become to replace the missing gene product. In case of gene inhibition therapy, the antisense gene inhibits the expression of the dominant gene.

Gene augmentation therapy

This is used to treat disorders caused by a mutation in a gene that prevents it from creating a useful product, such as a protein. This treatment reintroduces DNA into the cell that contains a functioning version of the deleted gene.

The new gene produces enough of a functional product to replace the protein that was previously absent. This is only effective if the disease’s consequences are reversible or if the body has not been permanently damaged. This may, for example, be used to treat diseases like cystic fibrosis by inserting a functional copy of the gene to fix the ailment.

Gene Therapy

Gene augmentation therapy process

Gene inhibition therapy

Infectious infections, cancer, and hereditary diseases due by abnormal gene activity can all be treated with this drug. The goal is to introduce a gene whose product either suppresses another gene’s expression or interferes with the activity of another gene’s product.

The goal of this treatment is to shut down a gene that promotes the proliferation of disease-related cells. The over-activation of an oncogene, for example, can result in cancer. It is thus conceivable to block further cell proliferation and stop cancer in its tracks by inhibiting the action of that oncogene by inhibition therapy.

Gene Therapy

Gene augmentation therapy process

Current Approaches

Transfer of a portion of DNA to any cell in the body that does not generate sperm or eggs is known as somatic gene therapy process. This side effects will not be passed down to the patient’s children. Transfer of a piece of DNA to cells that generate eggs or sperm is known as germline gene therapy process.

Somatic cell gene therapy

The non-reproductive (non-sex) cells of an organism are referred to as somatic cells. These are the cells of an organism other than sperm or egg cells, e.g., bone marrow cells, blood cells, skin cells, intestinal cells.

At present, all the research is directed to correct the genetic defects in somatic cells. In essence, somatic cell therapy involves the insertion of a fully functional and expressible gene into a target somatic cell to correct a genetic disease permanently.

Gene Therapy

Gene Therapy Process: Somatic cell gene therapy

Germ cell gene therapy

The reproductive (sex) cells of an organism constitute germ cell line. This therapy involving the introduction of DNA into germ cells is passed on to the successive generations. For safety, ethical and technical reasons, germ cell gene therapy process is not being attempted at present.

Gene Therapy

Gene Therapy Process: Germ cell gene therapy

The genetic alterations in somatic cells are not carried to the next generations. Therefore, somatic cell gene therapy process is preferred and extensively studied with an ultimate objective of correcting human diseases. A large number of genetic disorders and other diseases are currently at various stages of trials.

Current Approaches

The genetic alterations in somatic cells are not carried to the next generations. Therefore, somatic cell gene therapy process is preferred and extensively studied with an ultimate objective of correcting human diseases. A large number of genetic disorders and other diseases are currently at various stages of trials.

Further Readings

Reference