Fig. 3: Gene therapy using an Adenovirus vector. Source: Wikipedia (2009).
1.3.2 Gene Deletion This is the removal of an unwanted and undesired gene from an organism. Here, the restriction enzyme e.g.
endonuclease, is used to cut off the undesired gene. The sticky ends of the newly modified gene are allowed to
overlap with the vector and the new recombinant DNA is delivered to the host cell. 1.3.3. Gene Regulation/Termination This is used when we don‟t want to delete the gene. Here, an antisense messenger RNA (mRNA) of the gene is
synthesized and introduced into the cell, during protein synthesis, a sense mRNA is produced by the cell and
this combines with the antisense mRNA and protein synthesis is inhibited. Antisense molecule is a molecule
which binds specifically to a (sense) strand of RNA or to a DNA helix and stops it being used to code for a
protein. Antisense molecules are usually chemical derivatives of DNA or RNA. 1.4. Cases Where Genetic Engineering Has Been Applied Successfully The first biotechnology product approved for human health care was the synthetic human insulin which came
onto the market in the United States in 1982. Since then, more than 170 biotechnology related drugs and
vaccines have been approved by the United States Food and Drug Administration, ofwhich 113 are currently on
the market. Another 350 biotechnology medicines, together targeting over200 diseases, are in the later stages of
development. Amongst those approved during 2000 are medicines to treat pneumococcal diseases in children,
diabetes, cancer and haemophilia [2]. Genetic engineering has also been used in gene therapy. Gene therapy
involves the genetic engineering of a patient‟s genetic code to remove or replace a mutant gene that is causing
disease. There are two broad types of gene therapy that are possible. Germ line, or stem-cell, gene therapy
involves altering patients' DNA in their stem (reproductive) cells. The modification to their genetic “blueprint”
is permanent, and hereditary. This type of gene therapy is complex, and is considered too risky to undertake
until the underlying biology is better understood. It also raises many ethical concerns, for example, over the
potential misuse of the therapy to create “designer” babies. At the moment, germ-line gene therapy is banned in
many countries. The second type of therapy is somatic gene therapy. This involves engineering cells on a
“localized” basis, without affecting the patient‟s basic genetic “blueprint”. The first such therapy was approved
in 1990 to treat a four-year-old child suffering from severe combined immune deficiency (SCID). Some of the
child‟s white blood cells were extracted, genetically engineered in the laboratory and infused back into her
bloodstream. This successfully strengthened her immune system. Gene therapy techniques for cystic fibrosis
have also been approved, and candidate techniques for the treatment of Parkinson‟s disease, Alzheimer‟s disease
and some cancers are under development. Somatic gene therapy is likely to become very important for the
treatment of diseases caused by single mutant genes [2].