Gene therapy is the process of implanting DNA into a patient to remedy a genetic illness. To mitigate the effects of a disease-causing mutation, a functioning gene is usually incorporated in fresh DNA. Gene therapy uses segments of DNA to treat or prevent sickness.
To offset the consequences of a disease-causing mutant gene, the DNA is carefully selected. Although the method was developed in 1972, it has shown to be ineffective in curing human illnesses.
How does gene therapy work?
Now we will take a deeper look at what we mean by gene, how gene therapy technology works, how gene therapy works to treat genetic disorders.
Gene-
A specific sequence of nucleotides in DNA or RNA that controls the transmission and expression of one or more characteristics by dictating the structure of a particular polypeptide, particularly a protein, or directing the function of other genetic material and is generally found on a chromosome.
How does gene therapy technology work-
Gene therapy works by replacing or inactivating disease-causing genes. Gene therapy, in certain situations, involves the introduction of new genes into the body in order to treat a particular ailment. Doctors send a healthy copy of a gene to cells within the body via gene therapy. This healthy gene might be used to replace a mutated gene, inactivate a mutated gene, or even introduce a new gene.
Vectors, also known as carriers, deliver healthy genes to cells. The vectors are usually modified viruses that do not cause illness. Certain bacteria or circular DNA molecules may also operate as vectors (plasmid DNA). Additional ways for packaging and delivering genetic material, such as the use of nanoparticles, encapsulating lipid molecules, and using electric currents, are also being researched.
Vectors are introduced into the body by injection or intravenous (IV) infusion. In certain circumstances, physicians 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.
In gene therapy we do can go through procedures in the following ways-
Gene addition- It is a frequent gene therapy strategy being investigated for single-gene diseases—disorders in which one or both sets of your genes have been mutated. Using a vector, this gene therapy approach involves inserting functional (or healthy) copies of a gene (also known as a transgene) into a person’s cells.
In vivo or ex vivo, vectors convey the functional gene to the patient’s cells. Once within the cell, the transgene sends instructions to the cell that cause it to produce functioning proteins. The mutated gene does not need to be replaced or eliminated using gene addition therapy. This gives the cell the information it needs to make functioning genes rather than having to replace or eliminate the defective gene.
Gene editing- It uses a variety of ways to create specific breaks in DNA, with or without instructions to repair them.
Disruption/inactivation and correction/insertion are the two main strategies used in gene editing.
Genetic material that is responsible for the genetic disorder is “disrupted” or “inactivated.” This may be done by either turning off disease-causing genes or disrupting a gene that compensates for the disease-causing gene.
Creating a break in the gene and giving a correction template, or “inserting” new genetic material for the cell to utilise to fix the damaged gene.
How does gene therapy work to treat genetic disorders-
Gene therapy does the same in the following ways-
1.Replacing mutated genes- Some cells get ill as a result of specific genes functioning improperly or not at all. Replacement of faulty genes may aid in the treatment of some disorders. For example, the p53 gene inhibits the growth of tumours in most cases. Problems with the p53 gene have been related to a variety of cancers. If physicians could replace the cancer cells’ faulty p53 gene, the cancer cells may die.
2.Fixing mutant genes- Mutated genes that cause illness may be turned off so that they don’t promote disease any more, or good genes that assist prevent disease could be switched on to limit disease.
3.Making sick cells more visible to the immune system- Your immune system may not target diseased cells because it does not identify them as invaders in certain situations. Doctors may be able to employ gene therapy to teach your immune system to spot dangerous cells.
Conclusion-
In this article we have read about what we mean by gene, gene therapy, its procedures, how it is being used to treat genetic disorders. Also gene therapy is also being utilised to fix defects in dopamine production, such as in Parkinson’s disease, as well as to correct certain immune system issues and restore components required for proper blood cell activity in people with haemophilia and beta-Thalassemia. It’s also been used to regulate weight, height, and hair growth. Gene therapy techniques, like cosmetic surgery and nonsurgical treatments to improve skin, soft tissue, and body form, have presented ways to improve appearance.