Gene Therapy

Overview

Recall questions on Gene Therapy.

What you need to know

Answer each one before you reveal it.

Questions from this subtopic

Each one with the model answer underneath.

1. 1

   What is gene therapy?

   Model answer

   Gene therapy is the treatment of an inherited disorder by inserting a working copy of the gene into the patient's cells so that the missing protein can be made.

2. 2

   Distinguish between somatic and germ line gene therapy.

   Model answer

   Somatic therapy alters body cells only, so the change is not passed to the patient's children, whereas germ line therapy alters gametes or the zygote and would be inherited by every future generation.

3. 3

   Why is germ line gene therapy in humans not permitted?

   Model answer

   The change would be passed on to descendants who cannot consent, and any harmful side effect would become a permanent part of the population's gene pool.

4. 4

   Which protein is missing or faulty in muscular dystrophy, and what does it normally do?

   Model answer

   Dystrophin, which anchors the contractile machinery of the muscle fibre to the sarcolemma and prevents the fibre from being torn as it contracts.

5. 5

   What are the consequences for a boy with muscular dystrophy of having no functioning dystrophin?

   Model answer

   The muscle fibres are damaged every time they contract and are gradually replaced by fat and fibrous tissue, causing progressive weakness and wasting.

6. 6

   How is a healthy allele usually delivered into a patient's cells?

   Model answer

   It is packaged inside a modified virus, which has had its disease-causing genes removed and which then inserts the allele into the host cells it infects.

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