Recombinant DNA has opened new horizons in medicine

The developments in gene manipulation that have taken place in the last 30 years have
revolutionized medicine by increasing our understanding of the basis of disease, providing new tools for
disease diagnosis, and opening the way to the discovery or development of new drugs, treatments, and
vaccines.
The first medical benefit to arise from recombinant DN technology was the availability of
significant !uantities of therapeutic proteins, such as human growth hormone "#$#%, which is used to
treat growth defects. &riginally #$# was purified from pituitary glands removed from cadavers.
#owever, many pituitary glands are re!uired to produce enough #$# to treat 'ust one child. (urthermore,
some children treated with pituitary)derived #$# have developed *reutzfeld+,akob syndrome
originating from cadavers. (ollowing the cloning and e-pression of the #$# gene in E. coli, it became
possible to produce enough #$# in a .0)liter fermenter to treat hundreds of children. /ince then, many
different therapeutic proteins have become available for the first time. 0any of these proteins are also
manufactured in E. coli but others are made in yeast or animal cells and some in plants or the milk of
genetically modified animals. The only common factor is that the relevant gene has been cloned and over
e-pressed using the techni!ues of gene manipulation.
0edicine has benefited from recombinant DN technology in other ways "(ig. ...%. (or
e-ample, novel routes to vaccines have been developed1 the current hepatitis 2 vaccine is produced by the
e-pression of a viral antigen on the surface of yeast cells, and a recombinant vaccine has been used to
eliminate rabies from fo-es in a large part of 3urope. $ene manipulation can also be used to increase the
levels of small molecules within microbial or plant cells. This can be done by cloning all the genes for a
particular biosynthetic pathway and over e-pressing them. lternatively, it is possible to shut down
particular metabolic pathways and thus redirect intermediates towards the desired end product. This
approach has been used to facilitate production of chiral intermediates, antibiotics, and novel therapeutic
entities. New antibiotics can also be created by mi-ing and matching genes from organisms producing
different but related molecules in a techni!ue known as combinatorial biosynthesis. $ene cloning enables
nucleic acid probes to be produced readily, and such probes have many uses in medicine. (or e-ample,
they can be used to determine or confirm the identity of a microbial pathogen or to carry out pre) or peri)
natal diagnosis of an inherited genetic disease. 4ncreasingly, probes are being used to determine the
likelihood of adverse reactions to drugs or to select the best class of drug to treat a particular illness in
different groups of patients. Nucleic acids are also being used as therapeutic entities in their own right.
(or e-ample, antisense nucleic acids are being used to downregulate gene e-pression in certain diseases,
and the relatively new phenomenon of 5N interference is poised to become a breakthrough technology
for the development of new therapeutic approaches. 4n other cases, nucleic acids are being administered
to correct or repair inherited gene defects "gene therapy, gene repair% or as vaccines. 4n the reverse of
gene repair, animals are being generated that have mutations identical to those found in human disease.
These are being used as models to learn more about disease pathology and to test novel therapies.