Question

In part A, you analyzed genes that contribute to two diseases. (cystic fibrosis and muscular dystrophy) How can scientists use this information to develop new treatments for these diseases? Based on your findings, do you think that scientists will need to develop multiple treatments to control symptoms of these diseases? Explain your reasoning.

Answer 1

Answer:

By designing suitable gene therapies in order to restore target gene expression.

Explanation:

Cystic fibrosis and muscular dystrophy are inherited genetic disorders associated with serious health problems. Cystic fibrosis is caused by mutations in the gene that encodes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein, and it is a condition associated with abnormal production of sticky mucus that leads to problems in the lungs and the digestive systems. On the other hand, muscular dystrophy is produced by mutations in genes localized on the X chromosome such as, for example, the gene 'dystrophin'. Gene therapy is an experimental approach used to compensate abnormal gene function by introducing exogenous genetic material and thus restore their altered protein products. Consequently, personalized gene therapies can be useful to treat inherited disorders such as cystic fibrosis and muscular dystrophy.