Gene Therapy
Gene therapy describes a potential treatment/cure of inherited disorders/diseases.
The idea is that a normal version of the gene is inserted into the patient’s cells, so that a
functioning protein can be produced and the effects of the disease either cured or ameliorated.
In this exercise, you will select a genetic disease and explore the problems of trying to treat it with
gene therapy.
 Cystic Fibrosis
 Von Hippel-Lindau disease
 Haemophilia
 Parkinson’s disease
 SCID
 Stargardt disease
First, you need to pick a disease. The 6 disease/conditions listed above are all inherited; in other
words, they are the result of a genetic mutation.

Which one are you going to select for gene therapy research? And why?
You need to consider this from both a biological and commercial perspective. Is it possible? Is it
worth investing in?

For each disease, outline whether you think it is a suitable candidate for a gene therapy
development programme, and explain your decision.
Now choose a disease.
 What are the specific problems associated with providing gene therapy for this disease?
 How could you set about dealing with these problems?
 How will you measure success?
Much of the excitement around the potential of gene therapy is that it could provide a cure for
genetic diseases.
 Is this likely? Again, explain your answer.
A permanent cure could be achieved if gene therapy was carried out on an affected zygote.
 Explain why, and suggest why this type of gene therapy is currently illegal.
Gene therapy need not involve a functioning gene. For conditions caused by a dominant allele,
anti-sense RNA has been proposed as a possible therapy.
 Suggest what “anti-sense RNA” might be, and how it would work.