Gene therapy

Overview

Genes contain DNA, the essential code that governs much of the body’s form and function, influencing everything from hair color and height to vital processes like breathing, walking, and digestion. When genes malfunction, they can cause diseases, often due to mutations. Gene therapy aims to correct or replace faulty genes with healthy ones to cure diseases or enhance the body’s ability to fight them. This promising treatment targets a range of conditions, including cancer, cystic fibrosis, heart disease, diabetes, hemophilia, and AIDS.

The U.S. Food and Drug Administration (FDA) has approved gene therapy products for several conditions, such as cancer, spinal muscular atrophy, hemophilia, and sickle cell disease. However, for most people, gene therapy is accessible only through clinical trials. These research studies are crucial for determining the safety and efficacy of gene therapy approaches in humans, providing invaluable insights into how gene therapy affects the body.

Gene therapy is a medical technique that utilizes genetic material to prevent and treat diseases by altering your genetic makeup instead of relying on traditional treatments like medication and surgery. This approach allows healthcare providers to address the root cause of a disease or enable the body to produce beneficial medications or proteins. By transferring genetic material to your cells, gene therapy can change how cells produce proteins, potentially reducing disease-causing proteins, increasing the production of functional proteins, or creating new or modified proteins within cells.

Reasons for undergoing the procedure

The majority of gene therapies are now undergoing clinical trials. The development of safe and efficient therapies is significantly facilitated by clinical trials. Clinical trials are looking into the use of gene therapy to treat HIV/AIDS, some genetic disorders, cancer, macular degeneration, and other eye diseases.

The following purpose of gene therapy includes:

• Fixing the malfunctioned genes: It may be possible to deactivate disease-causing genes such that they no longer contribute to disease. Alternatively, the condition could be stopped by turning on good genes which help in disease prevention.
• Increasing the immune system’s awareness of unhealthy cells: The unhealthy cells are sometimes overlooked by the immune system, which keeps them from attacking them. To make your immune system perceive these cells as a threat, healthcare providers may utilize gene therapy.
• Replacing malfunction genes: Some genes malfunction or stop working completely, which causes diseases in some cells. Certain disorders may be treated by substituting healthy genes for unhealthy ones. For instance, the p53 gene typically stops tumor growth. Issues with the p53 gene have been connected to a number of cancer forms. The healthy gene may kill the cancer cells if medical practitioners could replace the defective p53 gene.

Risk

Gene therapy carries some potential risks. It is difficult to introduce a gene directly into your cells. Instead, a vector, which is a type of carrier, is typically used to deliver it.

Viruses are the most often used gene therapy vectors. This is a result of their ability to identify particular cells and insert genetic information into their DNA. Scientists modify the viruses, substituting disease-preventing genes for those that cause disease.

Risks associated with this method include:

• Unintentionally immunological response: The recently acquired viruses may be viewed as intruders by your body’s immune system. It might therefore attack them. Reactions from swelling to organ failure could result from this.
• Targeting the wrong cells: Multiple cell types can be attacked by viruses. It follows that cells other than those with malfunctioning cells could potentially become infected by the altered viruses. The sort of gene therapy utilized and its intended purpose determine the likelihood of harm to healthy cells.
• Infection caused by the virus: After the viruses enter the body, there’s a chance they could become disease-causing agents once more.
• Possibility of genetic errors: These errors can potentially lead to cancer.
• No guarantee: The field of gene therapy research is still in its early phases. These treatments do not promise a recovery from your medical condition or positive outcomes. It is possible that the treatment won’t work as planned or that it may have unanticipated negative effects.
• Complicated process: If your body receives foreign material, it may trigger an immunological response that keeps you out of clinical trials or future therapies.
• Long-term unknown effects: Gene therapy has effects that can last a lifetime, both positive and negative. The effects are uncertain because the science is so new.

Other vectors under study in clinical trials for delivering altered genes into the body’s cells include:

• Stem cells: Your body’s cells are all made up of stem cells. In order to create disease-fighting cells for gene therapy, stem cells can be changed or corrected in a lab.
• Liposomes: These particles can transport the new therapeutic genes to target cells and integrate them into the DNA of your cells.

Procedure

Genes that cause disease are replaced or made inactive using gene therapy. Gene therapy can occasionally introduce new genes into your body to address a specific medical condition.

Healthcare providers can introduce a functional copy of a gene into your body’s cells through gene therapy. This healthy gene can introduce a completely new gene, inactivate a mutant gene, or replace a damaged (mutated) gene.

The type of gene therapy used and the medical condition you have will determine which procedure you need. For instance, in a particular kind of gene therapy:

• A large needle may be used to remove bone marrow from your hipbone or draw blood.
• The required genetic material is then introduced to blood or bone marrow cells in a laboratory setting using a virus or another kind of vector.
• The cells are reinjected into your body through a vein or into tissue once the vector has entered the cells them in the lab. The modified genes are then absorbed by your cells along with the vector.

In a different kind of gene therapy, a viral vector is injected straight into an organ or the blood. Find out what kind of gene therapy will be utilized and what to anticipate by speaking with your healthcare provider.

Outcome

Gene therapy comes with the following advantages:

• Early treatment: Gene therapy can stop damage before it starts if you receive it early in your treatment. To what extent gene therapy can reverse damage is currently being studied.
• New and promising treatment alternative: Gene therapy offers hope for novel treatments for illnesses for whom there aren’t many other options at the moment. Many illnesses and disorders result in incapacity or early death if therapy is not received. Research indicates that certain illnesses’ progression has slowed down or even stopped with gene therapy.
• Targets the underlying cause of disease: Gene therapy enables the development of therapeutics that can target any gene in your body with precision.  [Text Wrapping Break]

Gene therapy has been used in human clinical studies to treat a number of diseases and conditions, such as:

• Blindness caused by retinitis pigmentosa.
• Cancer.
• Heart and blood vessel diseases.
• Hemophilia and other blood disorders.
• Infectious diseases.
• Inherited neurological disorders.
• Leukemia.
• Severe combined immunodeficiency.

However, a number of significant obstacles need to be overcome before certain forms of gene therapy may be considered a dependable kind of care, such as:

• Determining a dependable method for introducing genetic material into cells.
• Selected cells or genes to target.
• Reduced possibility of adverse reactions.

Despite the small number of gene therapy products available on the market, research into gene therapy is still ongoing in an effort to find new, efficient therapies for a range of illnesses.

Currently, studies are being conducted on the application of gene therapy to treat a number of diseases, including HIV/AIDS, cancer, eye abnormalities, and certain genetic disorders. We advise talking with your healthcare provider about the possibility of taking part in a clinical study involving gene therapy if you are interested in doing so. They can determine whether gene therapy is a viable treatment option for your specific illness.