1. What is gene therapy?

As the name suggest, gene therapy is a type of treatment technique, although 'genes' (DNA
segments) are used to cure diseases instead of medications or surgery. In gene therapy, scientists can do
one of many things. They can replace a gene that creates a medical condition with one that does not, add
genes to help the body fight or cure infection, or switch off trouble-causing genes.

Scientists use a vehicle called a "vector" that is genetically modified to carry the gene in order to
inject new genes directly into cells. Viruses, for example, are capable of delivering genetic information
into cells directly, and so can be used as vectors. It is however, modified to eliminate the ability to cause
an infectious disease before a virus can be used to carry therapeutic genes into human cells.

2. What are the forms of gene therapy?

Germline therapy and somatic gene therapy are the two forms of gene therapy. Although the type
of germ line is meant for permanent genetic modification of germ cells, somatic gene therapy refers to
gene repair of somatic or body cells.

Somatic gene therapy requires the introduction of a good gene into somatic cells, covering all
body cells except egg cells and sperm cells, with only the patient being treated as the end product. The
gene modification is temporary because it is just for the patient and is not inherited to the next generation.
It is a conservative treatment, and the side effects are limited only to the patient. In other words, while
any of the genes of the patient can be changed to cure a disease, the probability that the disease will be
passed on to the offspring of the patient won't change.

In germline cells, gene therapy has the ability to affect not only the person being treated, but also
his or her offspring. Because corrected gene is inherited to the next generations, through the sperm cell
and egg cell, and these germ cells are passed down by the parents to their descendants. If this gene
therapy continues to be successful, it can help prevent the transmission to the next generation of genetic
disorders, including those that arise in families.

3. What are the benefits of gene therapy?

Gene therapy, compared to drug therapy, may have a range of benefits when successful. For
example, in cancer. Some gene therapy types seek to improve the immune cells of the body to attack
cancer cells, while others are engineered to specifically attack cancer cells.

These therapies could minimize or remove the pain and discomfort that happens when a genetic
abnormality is found in the body of an individual. Almost 4 out of 5 illnesses that adversely influence
human health often have a genetic cause behind them. If doctors are able to replace or deactivate damaged
cells that have contributed to harmful diseases, so people and their families will be able to get the support
they need to live a more rewarding life.

The benefit of gene therapy can be long-lasting. While this value is not really applicable in every
case, in either the in-the-body or out-of-the-body approach, many patients may reverse their bothersome
symptoms in a brief period of time. There is also the probability that any of the changes will be inherited
to the next generation, thus minimizing the probability that they would suffer from a similar future.
4. What are the ethical issues on gene therapy?
 There is debate about the concept of germline gene therapy. While it could save future
generations from developing a specific genetic disease in a population, it could have an unexpected
impact on the growth of a child or have long-term side effects that are not yet understood.
Some people believe this type of treatment to be unnatural and compare it to "playing God."
Others are worried about the technological aspects. They fear that the genetic alteration that germ line
gene therapy transmits will potentially be harmful and dangerous, with the possibility for future
generations to have unexpected negative effects.
The difference between gene therapy for disease genes and gene therapy to modify desirable
features, such as height or hair and eye color, is sadly not clear-cut. No one will suggest that there are
good candidates for gene therapy for conditions that cause pain, injury, and, eventually, death. There is
however a thin line between what is called a "disease" in stable person, such as the dwarfism condition
achondroplasia, and what is considered a "trait" like having a short stature.
Many ethicists fear that there may be a "slippery slope" impact with respect to which genes are
used in possible gene therapy studies, as the success rate of germ line gene therapy increases and more
genes affecting various characteristics are found. In fact, the approval of germ line gene therapy may
contribute to the acceptance of gene therapy for genetic enhancement, which is feared by many.