Gene Therapy

Have you ever wondered about a world where we could solve each and every medical issue...The field of gene therapy can bring us closer to this ideal, perfect world. 

What is Gene Therapy? 

Gene therapy is an experimental form of treatment that modifies a person’s genes to treat or cure disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Gene therapy products are being studied to treat diseases including cancer, genetic diseases, and infectious diseases. 

How does Gene Therapy work? 

Gene therapies can work by several mechanisms:

• Replacing a disease-causing or mutated gene that causes disease with a healthy copy of the gene
• Inactivating, or “knocking out,” a disease-causing gene that is functioning improperly
• Introducing a new or modified gene into the body to help treat or fight a disease.

Constituents of Gene Therapy:

There are a variety of types of gene therapy products, including:

1. Plasmid DNA: Circular DNA molecules can be genetically engineered to carry therapeutic genes into human cells.

2. 
   

   Viral vectors: Viruses have a natural ability to deliver genetic material into cells, and therefore some gene therapy products are derived from viruses. Certain viruses are often used as vectors because they can deliver the new gene by infecting the cell. Once viruses have been modified to remove their ability to cause infectious disease, these modified viruses can be used as vectors (vehicles) to carry therapeutic genes into human cells. The viruses are modified so they can't cause disease when used in people. Some types of virus, such as retroviruses, integrate their genetic material (including the new gene) into a chromosome in the human cell. Other viruses, such as adenoviruses, introduce their DNA into the nucleus of the cell, but the DNA is not integrated into a chromosome.

3. Bacterial vectors: Bacteria can be modified to prevent them from causing infectious disease and then used as vectors (vehicles) to carry therapeutic genes into human tissues. The vector can be injected or given intravenously (by IV) directly into a specific tissue in the body, where it is taken up by individual cells. 

4. Human gene editing technology: The goals of gene editing are to disrupt harmful genes or to repair mutated genes. Alternatively, a sample of the patient's cells can be removed and exposed to the vector in a laboratory setting. 

5. Patient-derived cellular gene therapy products: Cells are removed from the patient, genetically modified (often using a viral vector) and then returned to the patient. The cells containing the vector are then returned to the patient. If the treatment is successful, the new gene delivered by the vector will make a functioning protein.

Is gene therapy safe? What are the ethical issues surrounding this field of research ?

Because gene therapy involves making changes to the body’s set of basic instructions, it raises many unique ethical concerns. The ethical questions surrounding gene therapy include:

How can “good” and “bad” uses of gene therapy be distinguished?

Who decides which traits are normal and which constitute a disability or disorder?

Will the high costs of gene therapy make it available only to the wealthy?

Could the widespread use of gene therapy make society less accepting of people who are different?

Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability? 

The idea of germline gene therapy is controversial. While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are yet unknown. Because people who would be affected by germline gene therapy are not yet born, they can’t choose whether to have the treatment. Researchers must overcome many technical challenges before gene therapy will be a practical approach to treating disease. For example, scientists must find better ways to deliver genes and target them to particular cells. They must also ensure that new genes are precisely controlled by the body. Due to these ethical concerns and procedural difficulties, the U.S. Government does not allow federal funds to be used for research on germline gene therapy in people. At this stage, it is being tested only in animal cells and other microorganisms.

Conclusion:

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique is still under study and has the potential to develop a lot more in the future.  

~by Kriya Shah.