1. What is Gene Therapy?

- Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure
disease or improve your body’s ability to fight disease. Gene therapy holds promise
for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease,
diabetes, hemophilia and AIDS.
2. What are some approaches to Gene Therapy?
a. Replacing mutated genes. Some cells become diseased because certain genes work
incorrectly or no longer work at all. Replacing the defective genes may help treat certain
diseases. For instance, a gene called p53 normally prevents tumor growth. Several types
of cancer have been linked to problems with the p53 gene. If doctors could replace the
defective p53 gene, that might trigger the cancer cells to die.

b. Fixing mutated genes. Mutated genes that cause disease could be turned off so that
they no longer promote disease, or healthy genes that help prevent disease could be
turned on so that they could inhibit the disease.

c. Making diseased cells more evident to the immune system. In some cases, your
immune system doesn't attack diseased cells because it doesn't recognize them as
intruders. Doctors could use gene therapy to train your immune system to recognize the
cells that are a threat.
3. What are the two types of Gene Therapy?
Germline therapy - Germline therapy involves the modification of the genes inside germ or
gamete cells, which include sperm or ova. Germline therapy would therefore be administered
during reproduction, where the modified gamete cells fuse to form a zygote. Once fused
together, the zygote divides and passes on the modified gene to all other cells of the body
during the development of offspring. In this way, germline therapy alters the genome of future
generations to come.
Although theoretically, germline therapy could counteract hereditary diseases, jurisdictions in
various countries such as Switzerland, Australia, and Germany prohibit the use of germline
therapy due to fears on the unknown risks of this therapy and whether it causes any long-term
effects in future generations. Germline therapy is also extremely expensive, which further limits
its practical use.
 a. Somatic gene therapy - Unlike germline therapy, somatic gene therapy involves the
insertion of therapeutic DNA into body cells, rather than germ cells or gametes. This
means that any effects of the therapy are confined to the individual being treated and
are not inherited by future offspring.
The field of somatic gene therapy is surrounded by fewer ethical issues as compared to
germline gene therapy. While this may be true, this therapeutic approach remains in the
early stages of development.
The first hurdle in somatic gene therapy is the successful incorporation of the new gene
into the genome. In fact, integrating the modified gene into the wrong part of the DNA
could induce rather than prevent disease. In addition to requiring the desired gene
needs to be expressed, the gene expression of the new gene needs to be regulated in
order to prevent over-expression that could also trigger disease.
4. What are Stem Cells?
Stem cells are the body's raw materials — cells from which all other cells with specialized
functions are generated. Under the right conditions in the body or a laboratory, stem cells
divide to form more cells called daughter cells.
These daughter cells become either new stem cells or specialized cells (differentiation) with a
more specific function, such as blood cells, brain cells, heart muscle cells or bone cells. No other
cell in the body has the natural ability to generate new cell types.
5. Give the ethical issues on Gene Therapy?
- Because gene therapy involves making changes to the body’s basic building blocks
(DNA), it raises many unique ethical concerns. The ethical questions surrounding
gene therapy and genome editing include:
 How can “good” and “bad” uses of these technologies be distinguished?
 Who decides which traits are normal and which constitute a disability or
disorder?
 Will the high cost 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?