Development in modern

genetics
TOPIC 8.4

Objectives
Discuss how the outcomes of the Human Genome Project are
being used in the development of new drugs and the social,
moral and ethical issues this raises.
Describe how drugs can be produced using genetically
modified organisms (plants and animals and micro organisms).
Discuss the risks and benefits associated with the use of
genetically modified organisms.

Human Genome Project

A genome is all of the DNA (or genes) of an organism.
The Human Genome Project was a multinational project
that determined the base sequence of the human
genome.

Development of targeted drugs

With the advances made by and from the HGP and the
science of proteomics, new disciplines within the field of
applied genetics have developed. These include:
1. bioinformatics based upon the use of computers to
store and analyse the huge amount of data that is
generated as the sequence of bases in different
genomes is discovered
2. pharmacogenomics analysis of the responses of
organisms to drugs, based on their genetic make-up

RNA interference (antisense technology RNAi), and

the suppression of cardiovascular restenosis
In antisense technology, tiny RNA molecules are
produced that are complementary to part of the base
sequence of a messenger RNA (mRNA) copy of a
particular target gene.
Thus the RNAi, once in position on the mRNA, will block
production of the protein the mRNA codes for.
This would otherwise have occurred in ribosomes. In
this way, a disease gene can indirectly be silenced, for
example.

 One successful trial

has
involved
an
antisense drug a
specific length of RNAi
that works by blocking
the action of the gene
responsible for the
proliferation events in
the
endothelium
there.
Trials of this drug
have
proved
successful.

Selective attack on cancer cells

In the search for new drugs that will target oncogenes with
the mutated K-Ras gene, research teams use paired cell
colonies (known as cell lines) that differ in only a single
mutated gene the K-Ras gene.
Using the cell colonies, thousands of drug compounds have
been screened.
So far, four compounds have been identified that distinguish
mutant cells from normal, healthy cells.
One of these compounds inhibits the growth of tumours in
mice, so it is the subject of further, thorough investigations.

Ethical issues
Who owns the information? Some groups have applied for patents
on genetic sequences so that they have ownership, or have to be
paid for any treatment developed using the knowledge of that
sequence.
Who is entitled to know the information about your genome if it is
sequenced/ Should insurance companies have access to the
information?
Will genetic screening lead to eugenics (the genetic selection of
humans) and designer babies?
Who will pay for the development of the new therapies and drugs/
Many possible highly specialised treatments may be very
expensive and will only be suitable for a few people.

The production of drugs using genetically modified

organisms

 Today a new type of genetic modification is also in use,

known as genetic engineering.
Genes from one organism are transferred to the set of
genes (the genome) of another unrelated organism. The
process is also known as recombinant DNA
technology.

The steps involved in genetic

engineering of E. coli for insulin
production.

Using transformed animals

Using transformed plants

Environmental and ethical issues of

genetic engineering