Genes are the essential physical and functional units of inheritance.

Genes are specific

series of bases that program instructions on how to make proteins. Proteins are very
important and carry out a most of life’s functions as well as making up bulk of cellular
structures. Genetic disorders occur when genes are altered, resulting in the encoded
proteins being unable to carry out normal functions. (gene therapy. Doc)

Gene therapy is a technique that involves inserting genetic material (DNA or RNA) into a
person’s cell to combat disease.. There is a variety of ways that researches can use in
order to correct ‘abnormal’ genes. This includes:

 A normal gene may be inserted into a nonspecific location within the genome to
replace a nonfunctional gene. This approach is most common.

 An abnormal gene could be swapped for a normal gene through homologous

recombination.

 The abnormal gene could be repaired through selective reverse mutation, which
returns the gene to its normal function.

 The regulation (the degree to which a gene is turned on or off) of a particular gene
could be altered.

Currently gene therapy is only being used on diseases that currently have no known cure.
In most gene therapy studies, a ‘normal’ gene is inserted into the genome to replace an
‘abnormal’ gene. A gene that is inserted directly into a cell does not usually function for
this reason a vector is used. A vector is a carrier molecule that is genetically engineered
to deliver the therapeutic gene to the cell. Certain viruses are often used as because they
can deliver the new gene by infecting the cell. However the viruses have evolved a way
of encapsulating and delivering their genes to human cells in a pathogenic manner.

Some of the different types of viruses used as gene therapy vectors:

 Retroviruses - A class of viruses that can create double-stranded DNA copies

of their RNA genomes. These copies of its genome can be integrated into the
chromosomes of host cells. Human immunodeficiency virus (HIV) is a
retrovirus.

 Adenoviruses - A class of viruses with double-stranded DNA genomes that

cause respiratory, intestinal, and eye infections in humans. The virus that
causes the common cold is an adenovirus.

 Adeno-associated viruses - A class of small, single-stranded DNA viruses that

can insert their genetic material at a specific site on chromosome 19.
  Herpes simplex viruses - A class of double-stranded DNA viruses that infect a
particular cell type, neurons. Herpes simplex virus type 1 is a common human
pathogen that causes cold sores.

In theory gene therapy may sound like simple way of curing disease without the use of
drugs or surgery, however through the clinical trials carried out on it seems to have raised
more questions then it has answered. A clinical trial is a research studies that test how
well new medical approaches work in people. Each study answers scientific questions
and tries to find better ways to prevent, screen for, diagnose, or treat of a disease.

Through gene therapy clinical trials it seems that in majority of cases success and failure
have fallen hand in hand. Clinical trials which have reported this result include:

One important clinical trial was conducted in 2006 where researches were able to shrink
tumors in 2 patients with melanoma, which was the first time since the development of
gene therapy that it had been used to successfully treat cancer.. The technique
researchers at the US National Cancer Institute used, involved taking normal
lymphocytes from the patients and infecting them with a retrovirus encoding a T-cell-
receptor (TCR) gene, which activates the lymphocytes into tumor recognition. For the
current study, the NCI researchers used genes encoding TCR specific for melanoma
antigens (MART-1). The researches at the NCI have also been able to isolate TCRs that
distinguish types of cancers such as breast, lung and other common cancers. Which
means this technique in treating advanced melanoma cancers brings hope to the fact that
this type of gene therapy could be utilized in different ways to combat many types of
cancer in the near future.

Although the media claimed that this was a ‘breakthrough’ in the world of gene therapy,
the claims of success also reared the risk associated with gene therapy. Although the two
patients, both male showed full clinical regression of metastatic melanoma, they were the
only two successful cases of a group of 17 patients. The other 15 showed no response
during the treatment period, 12 have since died and the remaining three have deteriorated
in condition.