8.3 other technologies

Gene therapy

Aims to treat or cure genetic abnormalities by identifying faulty genes and inserting healthy ones.

areas of possibility for gene therapy

Replacing mutated gene with a healthy copy. Fixing or inactivating mutated genes. Inserting a new gene that will fight the disease. Making the immune system recognise disease cells.

The concept of gene therapy (in relation to vectors)

Is that a vector can be used to deliver desired DNA into a cell. This DNA can be incorporated into the cells nucleus and undergo transcription and translation to produce the desired protein.

Type 1 diabetes Treatment possibility

reprogramming other cells to produce insulin. Gene for insulin is introduced into a vector , the vector is used to infect the desired cells, such as the alpha cells in the islets of langerhans, these cells incorporate the new DNA into their nucleuses and are able to use protein synthesis to produce insulin.

Cystic Fibrosis characterised

By sticky mucous secreted by the mucous glands. May clog the tiny air passages in the lungs and trap bacteria, making the person susceptible to infection. May cause irreversible lung damage and shorten life expectancy. Pancreas is also affected causing digestive problems.

CF and gene therapy

Is the logical choice for gene therapy treatment as it is a single gene disorder, and the most severely affected organ the lung is relatively easy to access. Disease is also slow to progress, this would enable gene therapy to occur before significant lung damage .

Huntingtons

Single gene disorder, gene therapy can be used to slow down or prevent its development.

Huntingtons symptoms

The mutated form of a protein called huntingtin results in nerve cells in the brain being damaged, causing physical, mental and emotional changes. The disease is characterised by occasional unintentional flailing movements of the limbs. The affected person also suffers from progressive dementia.

Huntingtons Research

Research in the US indicated that gene therapy for Huntingtons could be effective in humans.

Cell replacement therapy