Gene Therapy

About Gene Therapy

Gene therapy, also called gene transfer therapy, is a way to fix a genetic condition caused by a mutation by putting a healthy gene into a person’s genome. When a normal gene is put into the nucleus of a mutant cell, it most likely will not go to the same place on the chromosome as the defective allele. This may fix the mutation, but if the normal gene joins with another functional gene, it could cause a new mutation. There is a chance that the changed cells will continue to grow and make enough normal gene products to replace the mutant allele. This would make the whole body healthy again.

Methods Of Gene Therapy

People with cystic fibrosis, adenosine deaminase deficiency, familial hypercholesterolemia, cancer, and severe combined immunodeficiency (SCID) syndrome have tried gene therapy on somatic (body) cells. Even though gene therapy can lessen the symptoms of a disease, the change does not get passed on to the patient’s children. 

Germline gene therapy tries to make the germ line have better cells (e.g., cells of the ovary or testis). If it works, those cells will go through meiosis and make gametes as normal for the next generation. Animal tests have shown that experimental germline gene therapy works on animals but not on people. Researchers have also examined whether stem cell and gene therapy could be used together. In a pilot study of that method, skin cells from a patient with alpha-antitrypsin insufficiency were used. 

This genetic illness is linked to certain lung and liver disease types. They changed the cells’ programming to turn them into stem cells, fixed the wrong gene mutation, and then pushed the cells to grow into liver cells. The cells that had their genes changed and reprogrammed worked as expected.

The Way Gene Therapy Works

During gene therapy, genes that cause disease are changed or made dormant. Gene therapy sometimes treats certain illnesses by adding new genes to the body. Gene therapy allows doctors to put a healthy gene copy into a living cell. This healthy gene could add a new gene, turn off a bad (mutated) gene, or replace a gene already there.

Good genes get into cells through carriers, which are also called vectors. Most vectors are made from viruses that don’t spread disease. Also, other bacteria or round DNA molecules could be used as vectors (plasmid DNA). Researchers are also looking into the use of nanoparticles, the encapsulation of lipid molecules, and electric currents as ways to move and package genetic material.

Vectors are given to the body by injection or through the bloodstream (IV). In other cases, doctors will take cells from a patient and mix them with vectors in a lab. The vector-mixed cells are injected or given to the patient through an IV.

There Are Many Kinds Of Gene Therapy:

Dna Polyploidy: Human cells can get therapeutic genes from DNA molecules that have been changed genetically.

Virus Spreaders: Viruses are used to make some gene therapy products because they can naturally move genetic material into cells. Once viruses have been changed, so they don’t spread disease, they can be used as vectors to move therapeutic genes into human cells.

Bacterial vectors: Can change bacteria so they can’t spread diseases and then can use them to move therapeutic genes into human tissues.

Gene editing: The goal of gene editing is to replace broken or harmful genes.

Cellular gene therapy products that come from a patient: The patient’s cells are changed genetically (usually with a virus) and then returned.

Goals Of Gene Therapy And Guiding Principles

The goal of gene therapy is to fix bad genes that cause disease. It is done so that it can put normal genes into the nuclei of cells. So, a gene mutation is fixed, improved, replaced, or made up for in another way. To fix a congenital disability or a mistake in the genes, a new gene that works perfectly is put into the cells using more modern technology. 

The cells then change what they do. One strategy is to fix a broken gene so it can normally work again. Another is homologous recombination, which uses a normal gene to replace an abnormal gene. Another choice is to control the bad gene. Genetic manipulation of human body cells makes it possible to find better ways to treat cancer and other genetic diseases. Gene therapies of today focus on somatic, non-reproductive cells of the body, like those in the skin, muscles, liver, and bones. Even though this somatic gene therapy only works for one generation, it can fix genetic problems passed down from parent to child. 

Germline gene therapy, on the other hand, changes the cells that make eggs and sperm. Somatic gene therapy is the only gene therapy allowed in the U.S. Even though it has been tested on animals; it is not morally acceptable to use on people. By putting a bad gene into a fertilised egg, a new gene mutation could happen, which the baby would have when it was born. So, you might pass it on to the next generation. Enhancement gene therapy involves putting genes into an embryo or offspring that will make them grow taller or lose weight, depending on the goal. Eugenic gene therapy involves putting certain genes into a population to improve things like intelligence. People also think using these two gene therapies on people would be wrong.

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