GENE THERAPY

Gene therapy is when a DNA is introduced into a patient to treat a genetic disease. The
new DNA contains a functioning gene to correct the effects of a disease-causing mutation. It is
also an experimental technique that uses genes to treat or prevent disease.
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 is designed to introduce genetic material into cells to compensate for
abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to
be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to
restore the function of the protein.
A gene that is inserted directly into a cell usually does not function. Instead, a carrier
called a vector is genetically engineered to deliver the gene. Certain viruses are often used as
vectors because they can deliver the new gene by infecting the cell. 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.
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. Alternately, a sample of the
patient's cells can be removed and exposed to the vector in a laboratory setting. 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.

Types of Gene Therapy

1. Somatic gene therapy – transfer of a section of DNA to any cell of the body that
doesn’t produce sperm or eggs.
2. Germline gene therapy – transfer of a section of DNA to cells that produces sperm or
Eggs.

Categories of Gene Therapy:

1. Somatic-Cell gene therapy for the cure and prevention of diseases. Example :
insertion of a DNA sequence into a person’s cell to allow production of an enzyme
like adenosine deaminase.
2. Germ Line gene therapy for cure and prevention of diseases. Example: Insertion of an
adenosine deaminase sequence into early embryo or reproductive cells, which would
affect not only the individual but all of his or her offspring.
3. Somatic cell enhancement. Example: Insertion of a DNA sequence to improve memory
, increase height, or increase intelligence which would affect only the individual.
4. Germ line enhancement. Example: Insertion of a DNA sequence for enhancement into
a blastocyst, sperm or egg, which would affect future generation.
Gene Therapy Techniques

There are several ways by which gene therapy is administered. This includes the
following:

1. Gene augmentation therapy. It is designed for diseases caused by loss of function of a gene,
introducing extra copies of a normal gene may increase the amount of normal gene product to a
level where the normal phenotype is restored.

2. Gene inhibition therapy. This is suitable for the treatment of infectious diseases, cancer and
inherited diseases caused by the inappropriate gene activity. The aim is to introduce a gene
whose product either: inhibits the expression of another gene or interferes with the activity of
the product of another gene.

3. Gene therapy killing of specific cells. Genes are directed to the target cells and then
expressed so as to cause cell killing. Direct cell killing is possible if the inserted genes are
expressed to produce a lethal toxin(suicide gene) or a gene encoding a prodrug is inserted,
conferring susceptibility to killing by a subsequently administered drug.