Question
Some inherited genetic diseases were caused by the lack of an essential protein. Biotechnology may be used to treat individuals affected by these genetic diseases. Research into genetic diseases caused by the lack of an essential protein can focus on using biotechnology in two ways:
1. Produce the protein cheaply and affectively using microorganisms so that the patient can be regularly dosed with the protein.
2. Cure the affected person by inserting functioning genes so that the person is capable of producing the required protein.
Discuss difficulties that could occur when delivering a gene into a patient. These are things that researchers would need to consider, and check for, and ensure were not a problem before the patient could be said to be ‘cured’ and able to produce their own essential protein.
Answer
A faulty gene is isolated and replaced by a healthy/active gene. Isolation of gene of interest inserted into DNA of a suitable vector using vector using restriction enzymes and ligation and then cloned. Correct gene delivered back into the human, enabling coding of required protein.
Problems may arise with type of vector used, immune system may attack it/ may elicit an allergic response. Testing is needed to make sure the gene delivered works to correct cells and is incorporated into nucleus for protein synthesis. The quantity of protein made would have to be sufficient to effect a cure – needed to deliver enough gene to do this and genes must remain in patient long enough to have an effect.