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the elucidation of diseases that are multifactorial and involve a significant contribu-
tion from environmental factors. One of the best-studied examples of this type of
disease is cancer. Molecular genetic analysis has allowed a discrete set of cellular
genes, termedoncogenes, to be defined which play key roles in such events. These
genes and their proteins are also major points in the cell cycle and are intimately
invloved in cell regulation. A number of these are indicated in Table 6.8. In a number
of cancers well-defined molecular events have been correlated with mutations in these
oncogenes and therefore in the corresponding protein. It is already possible to screen
and predict the fate of some disease processes at an early stage, a point which itself
raises significant ethical dilemmas. In addition to understanding cellular processes
both in normal and disease states great promise is also held in drug discovery and
molecular gene therapy. A number of genetically engineered therapeutic proteins and
enzymes have been developed and are already having an impact on disease manage-
ment. In addition the correction of disorders at the gene level (gene therapy) is also
under way and perhaps is one of the most startling applications of molecular biology
to date. A number of these developments are indicated in Table 6.9.

Table 6.9A number of selected examples of targets for gene therapy


Disorder Defect Gene target Target cell


Emphysema Deficiency (a1-AT) a1-Antitrypsin (a1-AT) Liver cells


Gaucher disease
(storage disorder)


GC deficiency Glucocerebrosidase GC fibroblasts

Haemoglobinopathies Thalassaemia b-Globin Fibroblasts


Lesch–Nyhan syndrome Metabolic deficiency Hypoxanthine guanine
phosphoribosyl
transferase (HPRT)


HPRT cells

Immune system
disorder


Adenosine deaminase
deficiency

Adenosine deaminase
(ADA)

T and B cells

Table 6.10Current selected plant/crops modified by genetic
manipulation

Crop or plant Genetic modification
Canola (oil seed rape) Insect resistance, seed oil modification
Maize Herbicide tolerance, resistance to insects
Rice Modified seed storage protein, insect resistance
Soya bean Tolerance to herbicide, modified seed storage protein
Tomato Modified ripening, resistance to insects and viruses
Sunflower Modified seed storage protein

261 6.11 Molecular biotechnology and applications
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