Final answer:
To potentially cure cystic fibrosis, medical scientists should target and correct the DNA that codes for CFTR, by using gene therapy to introduce a correct version of the gene.
Step-by-step explanation:
To cure cystic fibrosis (CF), medical scientists should target the DNA that codes for CFTR. CF is caused by mutations in the DNA sequence of the CFTR gene, which result in the production of a defective CFTR protein. This defective protein is unable to function properly as an ion channel for transporting Cl- ions out of the cell, leading to the symptoms of CF. By correcting the underlying genetic mutation, the production of the normal functional CFTR protein would be restored, potentially curing the disease.
Additionally, the knowledge of the human genome allows for a better understanding of protein activity and the possibility of creating more specific drugs to target the defective CFTR protein. Gene therapy is being explored as a treatment method for CF, where a correct version of the gene is introduced to cells using a viral vector, potentially correcting the genetic mutation at its source. However, it is crucial to overcome various challenges associated with gene therapy, such as safe and efficient DNA delivery.