How Is Gene Therapy Used To Treat Genetic Disorders?
Gene therapy is a medical treatment or prevention method that involves fixing the underlying genetic condition. Instead of utilizing drugs or surgery, doctors can use gene therapy to treat an illness by changing a person’s genetic composition.
Gene therapy introduces new genetic material into a cell or cells of a living being, such as a human. It is a way of treating genetic disorders using the body’s own cells to produce a protein that is missing, another that is defective, or a different protein altogether. It works by correcting the genetic material inside a cell using genetic material from another cell. In mitochondria, for example, it replaces defective or missing genes or prevents the replication of faulty genes.
The first gene treatment approach, known as gene transfer or gene addition, was created to:
• Incorporate a new gene into cells to aid in treating a disease.
• Substitute a non-faulty copy of a gene for the disease-causing modified copy.
How does Gene Therapy work?
Gene therapy restores the functionality of key proteins by modifying the genetic code. Proteins are the cell’s powerhouses and the architectural foundation for the body’s tissues. A patient’s genetic code contains the instructions for generating proteins, and variations (or mutations) in this code can affect the synthesis or function of proteins that are important to how the body functions. Fixing or correcting for disease-causing genetic alterations may allow these crucial proteins to regain their function and the body to operate normally again.
In addition to the above, it is a powerful new technology that involves the introduction of new genes into cells to correct a genetic defect. The first gene therapy trials for sickle-cell anemia were primarily for children who were discovered to have the gene mutation during blood-screening tests.
Moreover, this type of genetic engineering relies on transferring genetic information, often from a donor cell, into a host cell. The goal is to use the information encoded by the foreign DNA to replace defective genes in the host cell to treat genetic disorders.
The types of Gene therapy products
•Plasmid DNA: Therapeutic genes can be genetically built into circular DNA molecules and delivered to human cells.
• Bacterial vectors: Bacteria can be engineered to prevent infection and utilized as vectors (vehicles) to deliver therapeutic genes into human tissues.
• Human gene-editing technology: The goals of gene editing are to disrupt harmful genes or repair mutated genes.
• Patient-derived cellular gene therapy products: Cells are taken from the patient, genetically altered (typically with the help of a viral vector), and then put back into the body.
Methods to Treat Genetic Disorders?
To treat genetic disorders, gene therapy uses various methods, including the use of viruses, cell therapy (to transfer genes into cells or the body to treat genetic disorders), or the administration of gene sequences that are corrected or replaced in the body.
What Kind Of Diseases Can Be Cured By Gene Therapy?
Most commonly, Gene Therapy is used to treat genetic diseases like Duchenne Muscular Dystrophy, Spinach disease, Hemophilia A, B, and C, and Cystic Fibrosis. It causes therapeutic proteins to be produced inside cells in order to treat a genetic disorder. Some treatments on the market, such as that of a group of boys with Duchenne Muscular Dystrophy, use gene therapy. Like other treatments, the goal is to introduce a healthy version of the gene that is causing the disease into cells to replace the faulty one.
The first gene therapy trials for sickle-cell anemia were primarily for children discovered to have the gene mutation during blood-screening tests.
Some Challenges Of Gene Therapy
• Getting the gene to the proper location and turning it on:
o It is crucial that the new gene reaches the right cell
o It would be inefficient to send a gene to the wrong cell, and it could also cause health concerns for the patient.
o Even once the correct cell has been identified, the gene must be activated.
o Cells can hinder this process by turning off genes that are active in odd ways.
What Are The Potential Risks Of Gene Therapy?
• The body’s immune system may react to the newly added therapeutic vector as if it were an intruder, just as it would to any virus. Inflammation and other hazardous consequences can result from an immune system reaction.
• An immune response could make gene therapy less effective if it works at all. As a result, prospective gene therapy patients are frequently screened with a simple blood test to see if they have antibodies to a particular virus.
• While the goal of utilizing a certain vector is to send a new gene to a specific tissue type, viruses can influence cells that aren’t targeted, causing harm or producing extra illness or disease.
Conclusion:
The goal of using Gene Therapy is to introduce a healthy version of the gene that is causing the disease into cells to replace the faulty one. The ability to mitigate the effects of disease or reverse conditions such as blindness, muscle weakness, and motor neuron disease has long been sought. Developments in gene therapy can transform the lives of patients living with incurable genetic conditions. However, this work has yet to be translated from the lab into a viable treatment for the whole human population, and the limited accessibility of traditional gene therapy techniques has prevented this.
While the purpose of gene therapy is to heal people suffering from a specific disease or condition, patient safety is paramount. Researchers, doctors, and the regulatory agencies that examine and approve all new gene treatments collaborate to assess a gene therapy’s safety and efficacy.
The treating physician and supportive care team provide meticulous monitoring and supervision throughout the gene therapy treatment procedure. They assist in the management of safety concerns during the healing process after gene therapy.
Patients and caregivers should always review their treatment plan with their physician, as well as the risks and advantages of specific gene therapy.