Types of Gene Therapy

Biotechnology

Biotechnology is focused on the large-scale production of biopharmaceuticals, including microorganisms, fungi, plants, and animals that have undergone genetic modification. Its applications include the fields of medicine and diagnostics as well as food processing, GM crops for agriculture, bioremediation, waste management, and energy generation. The main three crucial areas of biotechnology research are as follows:...

Applications of Biotechnology

Humans have used biotechnology to raise the standard of living, particularly in the areas of health and food production....

Gene Therapy

Gene therapy is a sort of medical care that alters a patient’s DNA in an attempt to cure or manage the disease. It involves the addition, deletion, or modification of particular genes in a person’s cells in order to stop the expression of undesirable genes or to increase the expression of favourable genes. Gene therapy can be used to treat a wide range of illnesses, including genetic disorders, some malignancies, and viral infections. It can be administered via viral or non-viral vectors. Gene therapy is being actively investigated and developed even though it is still in its early phases and shows enormous potential for the future of medicine....

Types of Gene Therapy

Somatic Cell Therapy: This occur mainly in the somatic cells of the human body. The only person who has the damaged cells will have healthy cells replaced because this is a single-person issue. Therapeutic genes are introduced into the somatic cells or stem cells of the human body using this technique. This approach of gene therapy is regarded as the most effective and secure one. This kind of gene therapy treats diseases like cancer, genetic abnormalities, and specific viral infections by focusing on the body’s non-reproductive cells. Germline Therapy: It occurs in the human body’s germline cells. This approach is typically used to correct genetic disease-causing gene variants that are handed down from parents to their offspring. The essential requirements are inserting functional DNA into the cells that generate eggs or sperm, the reproductive cells. Many countries do not permit sperm gene therapy because the dangers exceed the advantages. This kind of gene therapy is designed to be passed down from generation to generation and targets reproductive cells. Due to safety and ethical issues, it is still quite controversial and not yet extensively used. Gene Replacement Therapy: The goal of gene replacement therapy is to identify faulty genes, apply a correct copy of the gene’s DNA through a viral vector (sometimes referred to as a carrier molecule), and replace the detected faulty genes with the proper copies. In this kind of gene therapy, a patient’s cells are given a functioning copy of a gene that is absent or damaged. Gene Editing Therapy: Gene therapy includes gene editing. By using genetic material to treat or prevent disease, they both aim to tackle the underlying cause of the illness, such as a gene variant or mutation. Gene editing differs from gene therapy in that it delivers genetic material that can directly edit DNA fragments inside of a cell. This kind of gene therapy includes precisely altering a person’s DNA, such as by eliminating genetic mutations or introducing new genes. Gene Silencing Therapy: Gene silencing is a relatively new therapy method that makes use of the body’s natural processes to control the disease by silencing or inhibiting particular genes linked to particular diseases. Silencing here refers to temporarily suppressing a gene’s message that would otherwise result in an undesirable outcome. Through the use of methods like RNA interference, damaging genes’ expression is reduced in this type of gene therapy....

Application of Gene Therapy

By inserting a functioning copy of the missing or damaged gene, it can be used to cure inherited genetic illnesses such cystic fibrosis, haemophilia, and sickle cell anaemia. By introducing genes that either directly kills cancer cells or stimulate the immune system to target and eliminate them, it is possible to treat some cancers.  By adding genes that prevent virus replication, gene therapy can also be utilized to treat viral illnesses like HIV and hepatitis B. By introducing genes that create therapeutic proteins or by repairing damaged DNA, it is being looked at as a possible treatment for neurological illnesses, such as Alzheimer’s disease and Parkinson’s disease. With the introduction of genes that create therapeutic proteins or through the repair of damaged DNA in the eye, it is being studied as a possible treatment for ocular ailments, such as retinal diseases....

Advantages of Gene Therapy

As it explicitly targets the underlying genetic causes of an illness, it provides a tailored therapeutic strategy. Compared to medicines that only manage symptoms, this enables a more customized and efficient course of action. As the therapeutic gene can continue to produce its therapeutic effects in the patient’s cells for the duration of their life, it offers the potential to offer a one-time, long-lasting cure for some ailments. It provides a minimally invasive alternative to conventional therapies like organ transplantation or surgery. In some circumstances, it could eliminate the need for a lifetime of medications or other therapies. Instead of only treating symptoms, it has the ability to greatly improve a patient’s quality of life by restoring lost function or addressing the underlying cause of a condition. For uncommon disorders for which there may be no other treatments, gene therapy offers the potential to provide options. It is a branch of medicine that is developing quickly, and the creation of novel therapies and treatments may result in significant medical advancements and a greater comprehension of human genetics and disease....

Disadvantages of Gene Therapy

Delivering therapeutic genes to target cells is a complex procedure in the field of gene therapy, and there are numerous technological and scientific difficulties involved. Unintended effects of gene therapy could include the activation of dangerous genes, interruption of normal gene activity, or the development of malignancies. Immune responses to the therapeutic gene or the vector used to deliver it are another possibility. Since the therapeutic gene may not always enter or work effectively in the target cells, therefore gene therapy may not be effective for all individuals. The therapeutic impact could wear off quickly or the condition could return. Certain therapies including gene therapy can run into hundreds of thousands of dollars. Gene therapy is difficult to develop and commercialize due to the high cost of production and the small patient groups that potentially benefit from some treatments. Gene therapy is highly regulated, and bringing a gene therapy product to market can be a drawn-out and difficult procedure. Other ethical issues include the potential for unequal access to therapy based on social and economical criteria, the long-term implications of genetic editing, and other issues. Gene therapy raises moral concerns regarding the human genome’s manipulation, the possibility of unequal access to care based on social and economic circumstances, and the long-term repercussions of genetic modification....

Frequently Asked Questions

Question 1: What is Biotechnology?...