Applications of Gene Therapy

When DNA is implanted into a patient to treat a hereditary condition, this is known as gene therapy. A working gene is frequently included in the new DNA to remedy the consequences of a disease-causing mutation. 

To cure or prevent illness, gene therapy employs portions of DNA.The DNA is carefully chosen to counteract the effects of a disease-causing mutant gene. Although the approach was initially created in 1972, it has had minimal effectiveness in treating human ailments to date. Also we will learn about its types, applications along with its examples. 

Applications of gene therapy- 

Now we will take a deeper look at what we mean by genes, gene therapy, applications of gene therapy and examples of gene therapy applications. 

Genes- 

Chromosome’s main hereditary unit that takes a particular position. Each consists of nucleotides that are arranged in a linear pattern. Most genes code for a particular protein or part that results in a certain feature. 

Basically, we can say heredity’s basic, physical, and functional unit. DNA sequences are made up of genes (genotypes). The genotypes, in combination with other variables (such as environmental influences), define an organism’s phenotypic characteristic. Evolution and natural selection are fueled by changes in the DNA. Previously, the gene was known as a factor. Gregor Mendel was the first to discover the existence of a gene as a separate heritable element. 

Gene therapy- 

Human gene therapy aims to change the biological characteristics of live cells or adjust the expression of a gene for therapeutic purposes. 

Gene therapy is a strategy for treating or curing illness by altering a person’s DNA. Gene treatments may function in a variety of ways: 

 1.Putting a healthy copy of a disease-causing gene in place of the disease-causing gene 

 2.Inactivating a disease-causing gene that isn’t working as it should 

 3.For illness treatment, a new gene is injected into the body. 

Cancer, hereditary illnesses etc are in the list of those for which the treatment in gene therapy is    being researched. 

Gene therapy comes in a number of ways- 

 1.Plasmid DNA is a kind of circular DNA that may be genetically modified to deliver therapeutic genes to human cells. 

 2.Virus vectors have the inherent capacity to carry genetic information into cells, they are used in several gene therapy products. Viruses may be employed as vectors (vehicles) to transport therapeutic genes into human cells if they have been engineered to reduce their potential to cause infectious illness. 

 3.Bacterial vectors may be engineered to avoid infection and then utilised as vectors (vehicles) to deliver therapeutic genes into human tissues. 

 4.The aims of human gene editing technology are to disrupt dangerous genes or to fix damaged genes. 

 5.Cellular gene therapy products produced from patients: Cells are taken from the patients whose genes are changed (typically with the use of a viral vector), and then returned. 

Applications of gene therapy- 

Gene therapy is the process of changing the genes in your body’s cells in order to cure or prevent illness. 

In our DNA — the information that regulates much of the structure and function of your body, from growing you taller to controlling your bodily systems — is stored in your genes. Disease may be caused by genes that aren’t working correctly. 

 Gene therapy attempts to cure sickness or increase your body’s capacity to fight disease by replacing a broken gene or adding a new gene. Gene therapy has promise in the treatment of cancer, cystic fibrosis, heart disease, diabetes, haemophilia, and AIDS, among other disorders. 

Examples of gene therapy applications- 

Cystic fibrosis, haemophilia, muscular dystrophy, and sickle cell anaemia were among the first disorders to be treated using gene therapy, which focused on diseases caused by single-gene abnormalities.

There are three categories of disorders that may be treated by gene therapy: 

Firstly, Monogenic illnesses, in which a single locus (gene) is faulty and causes the disease, which is 100% heritable. Sickle cell anaemia, Severe Combined Immunodeficiency (ADA-SCID / X-SCID), Cystic fibrosis, Haemophilia, Duchenne muscular dystrophy, Huntington’s disease, Parkinson’s disease, Hypercholesterolemia, Alpha-1 antitrypsin, Chronic Granulomatous Disease, Fanconi Anaemia, and Gaucher Disease are examples of diseases.

Now polygenic diseases, in which numerous genes are implicated, and in which the illness is influenced by environmental and lifestyle variables. Heart disease, cancer, diabetes, schizophrenia, and Alzheimer’s disease are just a few examples and lastly infectious things like HIV/AIDS. 

Conclusion- 

In this article we have learned about what we mean by gene, gene therapy, different types of gene therapy, applications of gene therapy and diseases and disorders for which it is used. At last gene therapy has the potential to be a viable therapeutic option for a range of disorders in the near future. Individuals suffering from these illnesses (which may be remedied) may live longer, healthier lives free of symptoms and medical costs if certain hereditary defects can be repaired by gene substitution or alteration.