Gene Therapy: What is it? Is it Safe? Functions, Ethical Problems and Diseases Treated in the Present

The technique is used to treat or prevent diseases through genes.

It can allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of medication or surgery.

Researchers are testing several hypotheses for gene therapy, which include:

  • Replace a mutated gene that causes disease with a healthy copy of the gene.
  • Deactivate a mutated gene that malfunctions.
  • Modify DNA to help fight infection.

Although gene therapy is a promising option for treating several diseases, it is still risky and is still being studied to ensure safety and effectiveness.

Gene therapy is currently being tested only for diseases that do not have other cures, such as:

  • Hereditary disorders.
  • Some types of cancer
  • Certain viral infections.

Functions of gene therapy

It is designed to introduce genetic material into cells to compensate for abnormal genes or create a beneficial protein.

If a mutated gene lacks a necessary protein, gene therapy may be able to introduce a standard copy of the gene to restore the function of the protein.


A gene that is inserted directly into a cell usually does not work. Instead, a carrier called a vector is genetically engineered to release the gene.

Certain viruses are often used as vectors because they can release the new gene by infecting the cell.

The viruses chosen to participate in scientific studies are modified to prevent them from causing illness in people who volunteered for research.

These viruses integrate their genetic material (including the new gene) into a chromosome of the human cell like retroviruses. Other viruses, such as adenoviruses, introduce their DNA into the cell’s nucleus, but DNA is not integrated into a chromosome.

The vector can be injected or administered intravenously directly into a specific body tissue, which is captured by individual cells. Alternatively, a sample of the patient’s cells can be removed and exposed to the vector in a laboratory.

The cells that contain the vector are returned to the patient. Thanks to this new gene, a functioning protein will be produced by achieving successful treatment.

Researchers must overcome many technical challenges before genetic therapy is a practical approach to treating diseases.

For example, scientists must find better ways to administer genes and direct them to particular cells. They must also make sure that the body precisely controls the new genes.

Is gene therapy safe?

Gene therapy is under study to determine if it could treat diseases.

Current research is evaluating the safety of gene therapy; Future studies will test whether it is an effective treatment option.

Several studies have shown that this approach can have serious health risks, such as toxicity, inflammation, and cancer.

Because the techniques are relatively new, some of the risks may be unpredictable; however, medical researchers, institutions, and regulatory agencies are working to ensure that research in gene therapy is as safe as possible.

In the same way, laws, regulations, and regulations protect people who participate in research studies (called clinical trials).

What are the ethical problems surrounding gene therapy?

Because this therapy involves making changes in the basic instructions of the body, it raises many ethical concerns.

The idea of ​​germline gene therapy is controversial.

While it could prevent future generations of a family from having a particular genetic disorder, it could affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known.

Because people who would be affected by germline gene therapy have not yet been born, they can not choose whether they should receive the treatment.

What are the diseases that are dealing with gene therapy?

Gene therapy is currently available mainly in a research environment.

Hundreds of research studies are underway to test gene therapy as a treatment for genetic conditions, cancer, and HIV / AIDS.