Cystic Fibrosis and Genetic Mutation - Essay Example

This loss leads to lack of a single amino acid or phenylalanine at position 508. Though this is the most common mutation, many more have been identified to cause cystic fibrosis. In some cases, the CFTR protein is prevented entirely to take place at all and in some cases, CFTR protein does produce and get its way into the cell membrane but cannot operate properly. In patients where some CFTR reach the cell membrane to allow chloride transport, the severity of the disease is found less. One way of treatment method is to supply a different class of chloride channel to compensate for the loss of the CFTR chloride channel.

Gene therapy could be a better solution in the future in which a normal copy of the CFTR gene to the cells is delivered. In this way, direct synthesis of the normal CFTR protein will be possible to treat the biochemical abnormality leading to cystic fibrosis. Introducing healthy gene will take overall functions of the CFTR protein that are necessary including those which are yet not known. And the best way of gene therapy is to exploit the ability of viruses bringing their DNA to enter into cells.

Adenoviruses are suitable for this purpose due to their natural tendency to infect human airways.